Category Archives: Stem Cell Doctors

Sophomore saves sister’s life with stem cell donation – University of Miami

After a live-saving transplant to overcome leukemia, Sophia Nealon teamed up with her sister to create Bye, Bye, Bad Blood, a creative childrens tale that aims to teach young cancer patients about their diagnosis and prepare them and their families for what is to come.

For Sophia Nealon, saving her sisters life was by far the easiest decision she has ever made.

It took a simple swab from me and our entire family to see who had the highest DNA match percentage, said the Osprey, Florida, resident who studies chemistry at the University of Miami. l was a 100 percent match to be my sisters bone marrow transplant donor.

When the family traveled to New York City for a younger sisters birthday, it became obvious to them that something was wrong because Kassandra Nealon got sick again. Once back home in Florida, she went to see a doctor and the family got the devastating news. Kassandra Nealon, Sophias older sister and recipient of the life-saving stem cell transplant, was diagnosed with Acute T-Cell Lymphoblastic Leukemia at 23 years old, after becoming repeatedly ill with flu-type symptoms in late 2019.

They told her that she had two liters of fluid on her lungs, which was making regular tasks exhausting, said Nealon. Her doctors originally thought it was lymphoma but after testing her blood count, they shared with us that it was actually a type of leukemia. It was a big shock to everyone.

Following the diagnosis, the day-to-day routine of the Nealon family underwent a drastic change, as Kassandra Nealons appointments were located miles away in Tampa. Then the world entered a global pandemic. During this time, Nealon enrolled in her first semester as a University of Miami student, opting to complete the semester remotely.

My mom was the only one allowed at my sisters appointments, so it took a lot of coordination, but we figured out a system that worked for uslike staying in Airbnbs for some weekends and utilizing FaceTime to be with her during her chemo and radiation appointments, she said.

The type of cancer Nealons sister had is commonly diagnosed in young children and is rare in adults. After so many visits to various doctors offices, Kassandra Nealon noticed that there werent many books explaining the specific cancer that is faced mostly by adolescents.

Motivated by her friends and family, Kassandra Nealon felt compelled to create Bye, Bye, Bad Blood, a childrens book that lightheartedly explains the cancer to children and their families. Kassandra Nealon looked to none other than her sister, who draws as a hobby, to illustrate the book.

We decided to sell and market the book through Amazon because it would allow us to make the most profit, said Nealon, who had no prior knowledge about self-publishing a book.

That profit margin was important to Nealon and her sister because 100 percent of it goes directly to the Leukemia and Lymphoma Society,the largest voluntary health organization dedicated to fighting blood cancer in the world.

You dont know who youre helping out and how much you can change someones life with your donation, said Nealon, who also encourages the campus community to sign up with BeTheMatch.com to become a stem cell transplant donor. Becoming a donor is super easy and takes seconds of your time.

Nealons sister is now in remission. While promoting her book, she is determined to finish her medical school applicationsa process that was abruptly stopped as she battled cancer.

Those interested in donating a book to a childrens cancer treatment center or purchasing the book can visit byebyebadblood.com.

To learn more about stem cell transplants at Sylvester Comprehensive Cancer Center, visithttps://umiamihealth.org/en/sylvester-comprehensive-cancer-center/treatments-and-services/hematologic-(blood)-cancers-/bone-marrow-and-stem-cell-transplant.

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Sophomore saves sister's life with stem cell donation - University of Miami

Myelodysplastic Syndrome Treatment Options – Healthline

Your bone marrow produces immature blood cells, also called stem cells. These then develop into one of three types of mature blood cells:

If you have MDS, not all of these stem cells mature. As a result, immature cells can remain in the bone marrow or die. You will have lower levels of mature blood cells in your bloodstream.

A reduced mature blood cell count is a condition known as cytopenia, and its a main characteristic of MDS. A blood test known as a complete blood count (CBC) is one of the first diagnostic tests your doctor will order if they suspect you have MDS.

Removing bone marrow samples through aspiration and biopsy can also help your doctor better understand the nature of your blood marrow disorder. Once your doctor has diagnosed and treated your MDS, these tests can also show how well your bone marrow is responding to treatment.

You will often have a healthcare team if you have this type of disorder, which may include:

The team will develop a treatment plan thats partially based on the type of MDS you have. The World Health Organization (WHO) identifies six main types of MDS.

Your treatment plan may also be based on the prognostic score (outlook for survival) of the disease. MDS is different from most forms of cancer, which are grouped in stages and grades, because its scored on several factors, including:

Before you start MDS treatment, you might also consider your:

Next, well discuss each of the main treatment options for this group of diseases.

Supportive therapy is meant to treat MDS symptoms and prevent complications, rather than treat the underlying disorder itself. Supportive therapy is often used alongside other treatments.

Examples of supportive therapy include:

Lenalidomide is a type of medication called an immunomodulatory agent. Your doctor may suggest this oral medication if you have MDS and a certain genetic change known as an isolated del(5q) chromosome abnormality.

Lenalidomide helps boost red blood cell production in your bone marrow. Its designed to reduce your dependence on blood transfusions.

Experts in a 2017 research review called it an excellent option for MDS patients with low or intermediate disease risk. Potential side effects include:

Antithymocyte globulin is in a large group of drugs known as immunosuppressants that weaken the bodys immune response. Organ transplant recipients usually take them to help prevent rejection of the new organ. You may take antithymocyte globulin to keep your immune system from attacking stem cells in your bone marrow.

Immunosuppressant therapy may be appropriate for you if you have lower-risk MDS and havent had effective results with ESAs and transfusions. You might also try it if you have one or more autoimmune diseases.

One 2018 study found that antithymocyte globulin was effective in about 50 percent of the MDS cases studied.

Certain chemotherapy drugs, known as hypomethylating agents, activate specific genes in your stem cells to help them mature. Two examples of these agents are azacitidine and decitabine. These drugs are used when your doctor determines there is a serious risk for leukemia, which is a serious potential complication of MDS.

Chemotherapy can help improve blood cell counts, sometimes to the point where you no longer need transfusions, and reduce your risk of leukemia. Potential side effects include:

Other chemotherapy drugs may be given with the goal of killing atypical stem cells and allowing more of your healthy cells to mature.

While high-dose chemotherapy drugs can be very effective at sending MDS into remission, the side effects can be severe. You could experience a serious drop in white blood cells, and later, a greater risk of infections developing and progressing quickly.

A stem cell transplant involves removing some of your bone marrow, usually from the pelvic bone, and replacing it with bone marrow that produces healthy blood cells.

The procedure is usually reserved for more serious cases of MDS. Stem cell transplant is the closest treatment option to a cure, but its an invasive and challenging therapy. Because of this, stem cell transplant is limited to people who are considered healthy enough for both the procedure and the recovery process.

Doctors often use chemotherapy drugs with stem cell transplant to treat MDS. Together, they help support the growth of healthy blood cells to replace unhealthy or atypical cells.

While transplants are often very effective at achieving disease remission, particularly in certain patients, the Aplastic Anemia and MDS International Foundation says that a majority of MDS patients will see their condition return over time.

MDS can affect anyone at any age but generally affects adults ages 70 and older. According to the American Cancer Society (ACS), about 10,000 people are diagnosed with MDS in the United States per year.

Disease outcomes vary considerably. Its difficult to predict someones outcome because people respond differently to treatment.

ACS survival statistics range from a median survival of 8.8 years for people with a very low risk score to less than 1 year for MDS patients with a very high risk score.

These survival rates are based on data that includes years before treatments like chemotherapy were available. Its important to remember that researchers are continuing to develop new treatments that may improve disease outcomes.

You may have no symptoms early on or if you have mild MDS. However, for most people with the disease, anemia (low red blood cell counts) and symptoms such as chronic fatigue and shortness of breath are common.

If you have low white blood cell counts, the risk of serious infection may always be present. Low platelet counts can lead to easy bruising and bleeding complications.

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Myelodysplastic Syndrome Treatment Options - Healthline

Gold River Productions, Inc. (GRPS) Appoints Michael Berkowitz to spearhead the Rejuvenation Division with StemSpa – Yahoo Finance

Palm Coast, FL, Aug. 04, 2021 (GLOBE NEWSWIRE) -- After months of careful consideration, Gold River Productions, Inc. (OTC Pink: GRPS) (GRPS) opens its Rejuvenation Division and to welcome Michael Berkowitz as the newest member to the Gold River Team. His extensive background in the transformative field of cell-based therapy provides a significant pathway for sustainable revenue and directly compliments our new herbal formulations. With the coupling of our current product line and the considerable advances in stem cell therapeutic development, our direction brings viable attractive options to those suffering from an expansive range of degenerative conditions.

According to Fortune Business Insights, the global stem cells market is projected to grow from $11.90 billion in 2021 to $27.65 billion in 2028 at a CAGR of 12.8%. The factors contributing to the growth include increasing demand for biologics, technological advancement in pluripotential cell therapeutics, and a rising focus on the development of personalized medicines. The growing prevalence of chronic diseases worldwide is anticipated to boost demand through 2028.

As resourcing options are realized, the company intends to bring physical presence to these dynamic and unique options by offering a number of primary free-standing facilities to supply and educate our rapidly growing physician network.

Commenting as the newest member of the team, I am humbled by the support and dedication of Dr. Goulding and his associates at GRPS. Dr. Goulding shares my vision. Ive personally witnessed the effects that stem cells can have with appropriate administration and dosing in over 5000 cases, says Mr. Berkowitz, who will head the Rejuvenation Division of the GRPS subsidiary StemSpa. Ive worked with a number of universities and FDA approved manufacturers in recording and reporting data. Weve been involved in studies including M.S., joint issues, PTSD, and cancer. Im eager for the opportunity to continue and expand this with GRPS.

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Berkowitz continued, What intrigues me most about Dr. Bond and Dr. Goulding is how their products work in concert with pluripotential cells, and our InflammaplexTM formulation will serve as a foundation of support for the cells. Furthermore, almost anyone seeking relief will require adjunctive anti-inflammatory products for their pain and underlying issues. Empowering doctors with these unique adjuvants and giving their patients access to more preferable choices will be a game-changer.

Continuing, he stated, We are in negotiations to construct stand-alone facilities in strategic locations to establish StemSpa, which will serve as centers of excellence in the United States and offshore. We will begin with approximately 1000 doctors thanks to the relationship built between GRPS, heliosDx, and CEO Ashley Sweat of Rushnet (OTC Pink: RSHN). Im very excited to have doctors in this continuously expanding group utilize our inflammatory-reducing product line and have our technology at their fingertips. This will offer an amazing alternative to their patients as well as help us develop broad data collection and deliver data that these biologics are not only effective, but in the right hands and with the right techniques, possess endless possibilities to those suffering.

We are excited to have Mr. Berkowitz on board, says Richard Goulding, M.D., Chairman of the Board of GRPS. Mr. Berkowitz has been involved with over 5000 cases utilizing biologics and pluripotential cells and has a great deal of experience not only procuring appropriate cells, but in targeting specific concerns. The amount of video footage and anecdotal evidence is quite compelling. Theres a tremendous fit here, and the possibilities are exciting. Furthermore, we suspect that, with the proper base of nutrient support, we are optimizing the conditions for obtaining maximum efficacy with these techniques. We intend to be an industry leader for proper technique, procurement, and nutritional support in this complex field.

About Gold River Productions, Inc. Dedicated to changing peoples lives, Gold River is a groundbreaking company aiming at improving quality of life and longevity. Our diverse products and interests include nutraceuticals, CBD, land, education and more. With a unique staff of talented physicians, cultivators, and CBD experts, we are poised to tackle different disease states at an unprecedented level. Utilizing years of experience in herbals, rare cannabinoids, traditional and non-traditional methods of patient care, we are in the unique position to utilize CBD and herbals in the most effective ways possible. Emphasis on what already works, then augmenting effective formulas with high-quality cannabinoids in therapeutic levels, can achieve unprecedented symptom control in a myriad of disease states.

This document contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are subject to a number of risks and uncertainties, many of which are outside Gold Rivers control. These include but are not limited to the impact of competitors products, services and pricing; product demand; market acceptance; new product development; reliance on key strategic alliances; the regulatory environment; fluctuations in operating results; and other risks which are detailed from time to time in the Companys filings with the Securities and Exchange Commission and/or OTC Markets. Gold River Productions disclaims any obligation to update or alter its forward-looking statements whether as a result of new information, subsequent events or otherwise.

CONTACT: Gold River Productions, Inc. info@grpsinc.com http://www.GRPSInc.com

About heliosDx: heliosDX is a National Clinical Reference Laboratory offering High-Complexity Urine Drug Testing (UDT), Behavioral Drug Testing, Allergy Droplet Cards, Oral Fluids, Infectious Disease (PCR), and NGS Genetic Testing. We are contracted in 44 of the lower 48 states and looking to expand our reach and capabilities. Always staying ahead of the curve, by continually investing in our infrastructure with the most efficient scientific proven instruments, and latest cutting-edge software for patient and physician satisfaction. This allows heliosDX to provide physicians fast and accurate reporting meeting and exceeding industry benchmarks. We excel in patient and client care through physician designed panels that aid in testing compliance and reporting education.

Contact: Ashley Sweat asweat@heliosdx.com http://www.heliosdx.com (Under Development) @dx_helios

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Gold River Productions, Inc. (GRPS) Appoints Michael Berkowitz to spearhead the Rejuvenation Division with StemSpa - Yahoo Finance

Colin Jackson health: Im in constant pain Athlete to undergo stem cell therapy to help – Express

Colin Ray Jackson, CBE, 54, is a Welsh former sprinter and hurdling Olympic silver medal champion. Colins world record for his 60 metres hurdles stood for an incredible 27 years. As with most athletes of his calibre, Colin suffers with ongoing injuries from his sports days and will be undergoing a treatment to reduce the pain which Mike Tyson recently underwent too.

In recent years, stem cell therapy has been hailed as a miracle cure for many conditions, from wrinkles to spinal repair.

A stem cell is an immature, basic cell that has not yet developed to become, say, a skin cell or a muscle cell or a nerve cell.

There are different types of stem cells that the body can use for different purposes.

There is evidence that stem cell treatments work by triggering damaged tissues in the body to repair themselves, often referred to as regenerative therapy.

In animal studies, stem cell treatments have shown promise for various diseases, including heart disease, Parkinsons disease and muscular dystrophy.

A study undertaken by Dr Timothy McGuine found that 34 percent of athletes involved in the one-year study were more likely to report a history of knee and hip injuries.

Additionally, he found that specialised athletes, such as those competing in the Olympic games, were twice as likely to sustain a gradual onset or repetitive use injuries than athletes who did not specialise.

Dr McGuine also found that these athletes who find themselves competing year-round, stressing the same muscles and movements, and predisposed to the symptoms of burnout are at higher risk of long-term injuries.

Many doctors and athletes use stem cell therapy to treat sports injuries, such as Achilles tendinopathy or damaged knee ligaments, said Sports Health.

The site continued: While increasing in popularity, stem cell therapy is not considered standard practice by sports medicine doctors and not covered by most insurance companies.

The process of collecting stem cells is often called harvesting. Physicians usually harvest stem cells from the patients fat, blood, or bone marrow.

Many physicians who use stem cell therapy hypothesize that, when placed into a certain environment, stem cells can transform to meet a certain need.

Other sports stars who underwent stem cell therapy for long-term injuries included Cristiano Ronaldo, Rafael Nadal and most recently Mike Tyson.

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Colin Jackson health: Im in constant pain Athlete to undergo stem cell therapy to help - Express

Blood Drives To Be Held In NY In Effort To Save 4-Year-Old Boy With Leukemia – Mount Pleasant Daily Voice

08/01/2021 5:45 p.m.

A number of blood drives are being held in the area this month in the hope of saving a Long Island 4-year-old with leukemia.

Nassau County resident Ayden Paredes, of Glen Cove, was diagnosed with acute lymphoblastic leukemia on Feb. 7 of 2021, according to Mark Paredes, Ayden's father.

Ayden has been receiving chemotherapy treatments and blood transfusions since then, but doctors are now saying he needs a blood stem cell/bone marrow transplant to save his life, Mark said.

Mark said his son enjoys watching YouTube, playing video games, and also going to the beach and the park.

"He gets to do all this, but he gets very tired because his system is shot from the chemo treatments hes received since February," Mark said. "Hes a very inquisitive little boy, and we're just hoping to find a match for him."

Mark is asking members of the public to attend an event to see if they are a match to Ayden or thousands of other patients searching for a donor.

Mark said since Ayden is of mostly Hispanic descent, statistically, it is more difficult to find a match. He said it would be helpful if Hispanic members of the community could come out to see if they're a match.

Im very grateful and thankful to everyone who has been participating and registering," Mark added.

The blood drives will take place in New York and New Jersey in August in the hope of finding a match.

Donors have to be between ages 18 and 44. Appointments can be made online hereor by calling 800-933-BLOOD.

Here is a list of New York drives being held this summer:

FDNY Academy in Fort Totten Park Bayside, NY

10 a.m. 4 p.m.

* This is a donor drive in partnership with the FDNY. This is not a blood drive.

NYPD 13th Precinct Gramercy Park, Manhattan

10 a.m. 4 p.m.

NYPD Manhattan North 33rd Precinct Washington Heights, Manhattan

1 p.m. 7 p.m.

St. Patricks Church/K of C Glen Cove, NY

2 p.m. 8 p.m.

NYC Health Hospital/Jacobi Bronx, NY

8 a.m. 6 p.m.

Brentwood Legion Ambulance Brentwood, NY

1 p.m. 7 p.m.

BronxCare Health System Grand Concourse, Bronx, NY

11 a.m. 5 p.m.

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Blood Drives To Be Held In NY In Effort To Save 4-Year-Old Boy With Leukemia - Mount Pleasant Daily Voice

The Basics of MDS: Diagnosis and Staging – Curetoday.com

Myelodysplastic syndromes, known commonly as MDS, are a group of bone marrow diseases characterized by bone marrow failure, or an inadequate production of blood counts called cytopenia.

In a presentation at the CURE Educated Patient Leukemia Summit, Dr. Rami Komrokji, section head for Leukemia and MDS and Vice Chair of the Department of Malignant Hematology at Moffit Cancer Center, gave a run down on the diagnosis and staging process for MDS.

Diagnosing MDS

Komrokji explained that the myelo- prefix means bone marrow, and -dysplasia means abnormal-looking cells. When a patient has cytopenia, they may experience certain symptoms.

If patient is anemic, they will have shortness of breath, fatigue, palpitations, said Komrokji in an interview with CURE. If they have low platelets, they will have bleeding tendency, bruising. If they have low white blood cell counts, they will have maybe infections. So usually, either some of those symptoms will prompt blood testing, or on routine physical exam, the patients are found to have low blood counts. So that's usually the initial step.

Doctors will usually look into nutritional deficiencies such as B12, folate and ferritin, said Komrokji. Eventually, the patients will get a bone marrow aspirate and biopsy to diagnose their disease, which includes several parts.

There is the morphologic part, which means the pathologists are looking at the cells under a microscope, explained Komrokji. And then there is also some genetic testing. We look at cytogenetics nowadays, we look at gene mutations. So we put all of this information together to make the diagnosis.

The hematopathologist must see dysplasia, increased myeloblasts (immature cells known as blasts within the bone marrow) or certain cytogenetic abnormalities to make their diagnosis, Komrokji said.

Sometimes the diagnosis is straightforward, but sometimes it could be challenging, he added. It truly depends on an experienced hematopathologist to make the diagnosis.

Staging and Risk Stratification

Once a patient receives an MDS diagnosis, their doctor will go over risk stratification, or understanding what the risk of their disease is, which is what they consider staging, Komrokji said.

Now in MDS, its not like a lung cancer or colon cancer, he said. The disease does not spread around. The staging is based on the blood counts, on the percentage of those myeloblasts or immature cells (and) the chromosomal makeup of the cells. And nowadays, we sometimes also incorporate the presence of gene mis-happenings as well. So we get a lump score to estimate the risk.

Doctors typically use the International Prognostic Scoring System (IPSS) to categorize patients into one of five categories very low, low, intermediate, high and very high. The risk is the impact on survival and whether the disease will transform to leukemia, Komrokji explained. The disease risk must be known in order to tailor the patients treatment to them.

I always advise patients to see a specialized center in MDS, because obviously, those are not that common diseases, he said. A community oncologist could see a few (cases) per year, while an experienced center like in our place, we see like 15 to 20 per week.

Gene Mutations

Komrokji said that understanding gene mutations is an evolving field that is slowly becoming routine.

I advise all patients to inquire if theyve gotten genetic testing or not, he said. This sort of testing will help them understand any abnormalities. Doctors can look at a patients individual gene levels and detect for mutations, of which at least one was identified in 90% of patients with MDS.

Understanding the patients mutation(s) helps them tell whether there is a clonal hematopoiesis or mis-happening that occurred. It can also impact prognosis and allow them to further understand the disease risk.

And finally, some of them are targetable or important to follow through the treatment, said Komrokji. So patients should probably definitely have a genetic testing done. And sometimes after a treatment failure, we repeat it because we see other mutations that we could target with new drugs.

What Causes MDS?

In most cases, the cause of a patients MDS diagnosis is unknown.

We think it's phenomena of senescence or aging of those stem cells in the bone marrow that produces the blood, said Komrokji. Obviously, the process is very complicated. We have billions and billions of cells divide billions of times a day. So you know, as those cells age, mistakes can happen in them.

In most cases, he said, the mis-happenings which lead to the disease are random and at no fault of the patient. It is extremely rare for MDS to be inherited through familial genes.

There are, however, several known risk factors of MDS. If someone has history of another form of cancer and has received chemotherapy or radiation therapy, they may have possible stem cell damage and can develop MDS this is called therapy-related MDS. There has also been association of the disease with benzene exposure, chemical exposure and radiation exposure. Patients who have connective tissue diseases such as rheumatoid arthritis and lupus are at a slightly higher risk of getting MDS due to inflammation in the body and certain medications used to treat those diseases.

I would say there's a lot of better understanding of the disease in the past several years, of genetic mutation testing and incorporating them in practice, Komrokji said. And I think, you know, there are a lot of new treatments on the horizon for patients; there are several clinical trials in advanced phase.

For more news on cancer updates, research and education, dont forget tosubscribe to CUREs newsletters here.

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The Basics of MDS: Diagnosis and Staging - Curetoday.com

Pharming Group reports financial results for the first half of 2021 – PRNewswire

Recovery continued into Q2 2021 as patient enrollment and product demand increases

LEIDEN, The Netherlands, Aug. 5, 2021 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM/NASDAQ: PHAR) presents its preliminary (unaudited) financial report for the first six months of 2021 ended June 30, 2021.

Financial Summary

Amounts in US$m except per share data

H1 2021

H1 2020

% Change

Income Statement

Revenues

93.2

97.8

(5)%

Gross profit

83.8

86.9

(4)%

Operating profit

17.2

35.7

(52)%

Profit for the year

14.4

20.3

(29)%

Balance Sheet

Cash & marketable securities

189.8

173.8

9%

Share Information

Basic earnings per share (US$)

0.022

0.032

(31)%

Diluted earnings per share (US$)

0.019

0.028

(32)%

Financial highlights

Operational highlights

Post-period operational highlights

Chief Executive Officer, Sijmen de Vries, commented:

"As expected, revenue growth during Q2 2021 has continued to recover, following the impact of COVID-19 on Q1 2021, as underlying demand and patients benefiting from RUCONEST treatments for their HAE increases. We are confident this positive trend will continue for the remainder of the year and, supported by our strong cash position, will enable us to continue our planned investment in R&D and the ongoing preparations for the launch of leniolisib, which, subject to regulatory approval, is on track for the end of 2022 following the completion of patient enrollment in the potentially registration enabling study in APDS.

In our earlier pipeline, we initiated enrollment of patients in a multi-center Phase IIb clinical trial of rhC1INH for the prevention of acute kidney injury after myocardial infarction. In addition, post period, we delivered on one of our strategic objectives to strengthen our longer-term HAE pipeline, through a collaboration with Orchard Therapeutics, to develop and commercialize the pre-clinical ex-vivo autologous hematopoietic stem cell therapy product OTL-105, which has the potential to become a curative treatment for HAE. We remain focused on the positive progress against our three-pillar strategy of sales, R&D and acquisitive growth."

Outlook

For the remainder of 2021, we expect:

No further specific financial guidance for 2021 is provided.

About Pharming Group N.V.

Pharming Group N.V. is a global, commercial stage biopharmaceutical company developing innovative protein replacement therapies and precision medicines for the treatment of rare diseases and unmet medical needs.

The flagship of our portfolio is our recombinant human C1 esterase inhibitor (rhC1INH) franchise. C1INH is a naturally occurring protein that down regulates the complement and contact cascades in order to control inflammation in affected tissues.

Our lead product, RUCONEST, is the first and only plasma-free rhC1INH protein replacement therapy. It is approved for the treatment of acute hereditary angioedema (HAE) attacks. We are commercializing RUCONEST in the United States, the European Union and the United Kingdom through our own sales and marketing organization, and the rest of the world through our distribution network.

In addition, we are investigating the clinical efficacy of rhC1INH in the treatment of further indications, including pre-eclampsia, acute kidney injury and severe pneumonia as a result of COVID-19 infections.

We are also studying our oral precision medicine, leniolisib (a phosphoinositide 3-kinase delta, or PI3K delta, inhibitor), for the treatment of activated PI3K delta syndrome, or APDS, in a registration enabling Phase 2/3 study in the United States and Europe.

Furthermore, we are leveraging our transgenic manufacturing technology to develop next-generation protein replacement therapies, most notably for Pompe disease, which is currently in preclinical development.

Forward-looking Statements

This press release contains forward-looking statements, including with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, Pharming's ability to overcome the challenges posed by the COVID-19 pandemic to the conduct of its business, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2020 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2020 filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release.

Inside Information

This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation.

For further public information, contact:Pharming Group, Leiden, The NetherlandsSijmen de Vries, CEO: T: +31 71 524 7400 Susanne Embleton, Investor Relations Manager: T: +31 71 524 7400 E: [emailprotected]

FTI Consulting, London, UKVictoria Foster Mitchell/Alex Shaw T: +44 203 727 1000

LifeSpring Life Sciences Communication, Amsterdam, The NetherlandsLeon Melens T: +31 6 53 81 64 27 E: [emailprotected]

Conference call dial-in informationThursday August 5, 2021 13:00CET/07:00ETPlease note, the Company will only take questions from dial-in attendees.

Dial-in details:

Netherlands (Local) 085 888 7233

United Kingdom 0800 640 6441

United Kingdom (Local) 020 3936 2999

All other locations +44 20 3936 2999

Access code: 914296

Webcast Link:https://webcast.openbriefing.com/pharming-aug21/

Pharming Group N.V.Condensed Consolidated Interim Financial Statements in US Dollars (unaudited)For the period ended 30 June 2021

Appendix: Main condensed consolidated Interim Financial Statements reported in Euros

(This appendix is not part of the Condensed Consolidated Financial Statements)

CONDENSED CONSOLIDATED STATEMENT OF PROFIT AND LOSS

For the 6-month period ended 30 June

Amounts in $ '000

notes

HY2021

HY 2020

Revenues

7

93,237

97,827

Costs of sales

8

(9,487)

(10,885)

Gross profit

83,750

86,942

Other income

1,354

525

Research and development

(24,206)

(17,658)

General and administrative

(15,060)

(9,846)

Marketing and sales

(28,686)

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Pharming Group reports financial results for the first half of 2021 - PRNewswire

Blood donation clinic will honour life of Jocelyn McGlynn – Chatham Daily News

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A blood donation clinic is being held Aug. 10 in Chatham to honour the life and memory of the late Jocelyn McGlynn, whose courageous battle with leukemia inspired many to register to become stem cell donors.

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A blood donation clinic is being held Aug. 10 in Chatham to honour the life and memory of the late Jocelyn McGlynn, whose courageous battle with leukemia inspired many to register to become stem cell donors.

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McGlynn, who was a medical science student at Western University in London with dreams of becoming a doctor, became an advocate for stem cell and blood donations while she battled leukemia, which took her life on Aug. 15, 2020, at age 23.

McGlynn was first diagnoses in the fall of 2018 when she went to see doctors for what appeared to be a cold she could not shake. This was the beginning of a tough medical battle that included multiple rounds of chemotherapy, radiation and two bone marrow transplants.

Joc was selfless and strong, caring and courageous, wonderful and wise, said McGlynns mother, Jacqueline McGlynn, about her daughter becoming a vocal champion for those fighting similar medical battles.

She loved to live and did not want others to suffer as she did.

During her own fight, Jocelyn McGlynn continued to encourage stem cell and blood donations, which included taking part in Walk the Night for the Leukemia and Lymphoma Society of Canada and creating awareness videos for Blood Sweat Spin and Canadian Blood Services.

McGlynn used her creative talents to envision and bring to reality her Lets All Go Get Swabbed music video. She wrote the lyrics, played every note, directed, edited and starred in the video while recovering from chemotherapy.

People can help continue McGlynns efforts by scheduling an appointment for the upcoming blood donor clinic being held Tuesday at the Chatham YMCA from noon to 7 p.m. Donations can be booked online at blood.ca, through the GiveBlood App or by calling 1-888 2 DONATE (236-6283).

A single donation of blood is equal to one unit of blood, and a leukemia patient can require up to eight units of blood per week, stated the release.

As McGlynn wrote: Everybody join the team, theres a cure in your bloodstream.

Maureen Macfarlane, event co-ordinator with Canadian Blood Services, said there is an increased need for blood donations as hospital procedures, which were previously on hold during the COVID-19 pandemic, are being scheduled.

Another blood donor clinic is also being held in Chatham on Aug. 31 from 1 p.m. to 7 p.m. at the Retro Suites Hotel.

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Blood donation clinic will honour life of Jocelyn McGlynn - Chatham Daily News

Symptoms of seizures: Early signs, post-seizure, and more – Medical News Today

A person having a seizure may experience muscle spasms, blink rapidly, fall to the ground, or lose consciousness. They will require medical diagnosis of and treatment for the underlying cause of seizures.

Seizures may occur as a side effect of a health condition that originates from the brain, such as epilepsy.

Nonepileptic seizures may occur as well. These vary in type and may affect individuals with curable or reversible health conditions.

Other catalysts for seizures include:

This article will discuss in further detail the symptoms and types of seizures, their causes, the aftereffects, and more.

Sometimes, people will experience certain sensations, called an aura, prior to a seizure. These symptoms result from electrical changes in the brain that occur when the seizure starts.

They act as a warning that a seizure will happen. Yet the exact symptoms will depend on which part of the brain the seizure starts in.

According to John Hopkins Medicine, an aura typically occurs before a focal seizure, especially a complex focal seizure. The most common aura involves feelings such as:

People may also experience visual changes, hearing abnormalities, or changes in their sense of smell prior to having a seizure.

If a person notices any of these symptoms, they may want to keep track of them. Recording details of symptoms and when they happen can help a doctor diagnose and treat seizures.

Symptoms can vary depending on the type of seizure an adult has, but they may include:

Seizures may last a few minutes. However, the Centers for Disease Control and Prevention (CDC) encourage people to call 911 if:

Babies can also have seizures. However, a person may have more difficulty recognizing them because a babys healthy, typical responses may look similar to symptoms of a seizure.

Newborns commonly jerk, twitch, or contort their faces into grimacing expressions. So, a person can easily mistake these movements for seizures or not recognize them as seizure symptoms at all.

Seizure symptoms may include any or all of the following:

According to the University of Chicago Medicine, seizures are particularly likely if a baby repeats the same movements for approximately the same duration at the same time of day, usually when waking up or going to sleep. For babies prone to seizures, symptoms commonly occur every day.

In general, research suggest that genetics play an important role in whether a baby will experience seizures.

According to a 2018 research review, one-third of children with febrile seizures have a family history of seizures. The risk of a baby having febrile seizures is around 20% with an affected sibling and 33% with affected parents.

After a seizure, a person may experience a variety of effects that may persist for several minutes or even hours.

According to the National Institute of Neurological Disorders and Stroke, people may feel tired, weak, and confused after a seizure has ended. Other common symptoms include headaches or pain in the muscles that contracted during the seizure.

The specific aftereffects that a person experiences can help a healthcare professional determine which part of the brain seizure originated in.

One example is Todds paralysis, a temporary paralysis in part of the body due to the area of the brain the seizure originated in. If the seizure stemmed from the temporal lobe, a person is more likely to experience language or behavioral changes.

According to the CDC, two main types of seizures exist: focal onset and generalized onset.

Generalized seizures affect both sides of the brain, and they include absence seizures and tonic-clonic seizures. These types of seizures produce many common seizure symptoms, such as:

Focal seizures, or partial seizures, originate in just one area of the brain. Subcategories of focal seizures include:

People with epilepsy may experience all these types of seizures.

Psychogenic nonepileptic seizures may also occur, due to psychological distress. Though these are not epileptic seizures, they have similar symptoms.

These symptoms include:

Research suggests the most common cause is a conversion disorder. This occurs when a person has the symptoms of a health condition without an actual underlying condition or injury.

Nerve cells in the brain send electrical signals to communicate. Any change to this electrical activity can cause seizures. Factors that can cause abnormal electrical activity include:

According to a 2021 article, genetics play a role in whether a person will experience epilepsy. The article also lists other potential causes for seizures:

With a treatable underlying cause, such as hypoglycemia, seizures are not likely to reoccur after treatment.

For epilepsy specifically, the CDC states that the condition is most likely to develop in children under age 2 and adults over age 65.

Not every seizure is an emergency, and while they can be fatal, the CDC states that most people can live safely with seizures. Seizures may turn life threatening if:

Although very rare, sudden unexpected death in epilepsy (SUDEP) does occur. This refers to deaths that have no other cause than the seizure itself.

SUDEP is the cause of death in roughly 1.16 out of every 1,000 people with epilepsy.

If a person experiences a seizure for the first time, they will need prompt medical attention. A doctor can then diagnose the underlying cause and suggest appropriate treatment.

If a person experiences subsequent, more severe seizures, they should also seek medical help. A doctor can analyze patterns, discuss any aftereffects, and reevaluate treatment to help reduce the severity of seizures.

People who experience seizures may find it helpful to record the details of each episode to report to their doctor.

If people witness someone having a seizure, they will need to call 911 for any seizure that lasts longer than 5 minutes, according to the CDC, or if any injury occurs during the seizure.

Seizures may be epileptic or nonepileptic, and they stem from a disruption in nerve cell activity in different parts of the brain. Seizures typically last for only a few minutes at a time.

A person may experience various symptoms before, during, and after a seizure.

Tracking specific symptoms of a seizure may help a doctor diagnose or adjust treatment for seizures. Learning to recognize the warning signs of a seizure can also help people get into a safe space before a seizure occurs.

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Symptoms of seizures: Early signs, post-seizure, and more - Medical News Today

Lymph nodes: Purpose, location, and disease warning signs – Medical News Today

Lymph nodes are small, bean-shaped glands that play a crucial role in the immune system. They filter lymphatic fluid, which helps rid the body of germs and remove waste products.

The body contains hundreds of lymph nodes. They form clusters around the body and are particularly prominent in areas such as the neck, armpit and groin and behind the ears.

The bodys cells and tissues dispose of waste products in lymphatic fluid, which lymph nodes then filter. During this process, they catch bacteria and viruses that could harm the rest of the body.

Lymph nodes are an essential part of the bodys immune system. Due to their function, they come into contact with toxins, which can cause them to swell. Although swollen lymph nodes are common, they may occasionally indicate lymph node cancer, or lymphoma.

Keep on reading to learn more about lymph nodes and their function within the immune system.

Lymph nodes are part of the lymphatic system, which is a complex network of nodes and vessels.

In certain areas of the body, such as the neck, armpit, and groin, lymph nodes sit close to the skin. This means a person may feel them swell when an infection develops.

Lymph nodes are also present in the stomach and between the lungs. However, there are no lymph nodes in the brain or spinal cord.

The name of a lymph node depends on its location in the body.

Lymph nodes form clusters throughout the body. Their main function is to filter out potentially harmful substances.

All tissues and cells in the body excrete lymphatic fluid, or lymph, in order to eliminate waste products. The lymph then travels through vessels in the lymphatic system and passes through lymph nodes for filtering.

Lymph nodes contain lymphocytes. These are a type of white blood cells that help destroy pathogens, such as bacteria, viruses, and fungi. When lymph nodes detect a pathogen in the lymph, they produce more lymphocytes, which causes them to swell.

Upon encountering bacteria or damaged cells, lymph nodes destroy them and turn them into a waste product.

When the lymph reenters the bloodstream, waste products travel to the kidneys and liver. The body then excretes waste products in the urine and feces.

Learn more about how the lymphatic system works here.

Swollen lymph nodes do not always indicate cancer. Below, we list some of many conditions that can cause lymph node swelling.

Lymphadenitis occurs when bacteria, viruses, or fungi in the lymph infect lymph nodes. When this happens, lymph nodes swell and are painful to the touch.

If multiple clusters of nodes become infected, a person may feel pain and swelling in both their neck and groin.

The most common type of lymphadenitis is localized lymphadenitis. This means the condition only affects a few nodes. If the infection occurs in several node clusters, a doctor will likely diagnose generalized lymphadenitis.

The condition usually results from an infection elsewhere in the body.

Symptoms of lymphadenitis include:

Lymphadenitis treatments include:

The type of treatment necessary will depend on a variety of factors, such as the severity of the disease and a persons underlying conditions and allergies. A doctor will help a person choose the most suitable treatment based on these factors.

Learn more about swollen lymph nodes in the neck here.

Swollen lymph nodes in the neck may be due to a viral or bacterial throat infection, such as strep throat.

Viral throat infections, such as colds, can present with swollen lymph nodes, a runny nose, and pinkeye.

These infections usually resolve on their own. However, a person can take over-the-counter pain relievers to alleviate pain they may experience when swallowing.

Strep throat is a bacterial infection that develops in the throat and tonsils due to group A streptococcus. People may contract strep throat if they come into contact with droplets containing the strep bacteria.

A person with strep throat may experience swollen lymph nodes on the neck, a sore throat, a fever, and red spots on the roof of the mouth.

Doctors treat strep throat with antibiotics.

Impetigo is an infection that develops due to group A streptococcus and may cause lymph nodes in the armpits and groin to swell.

A person can contract impetigo when the bacteria enter the body through a break in the skin. This can happen through sharing a towel, razor, or yoga mat.

Symptoms of impetigo include:

If a person has impetigo, they should seek medical attention to address their symptoms and prevent the condition from spreading to others.

Treatment will usually involve antibiotics.

Ringworm, or jock itch, is a fungal infection that can affect many areas of the body. If the fungus develops in the groin, a person may experience lymph node swelling in that area.

Typically, ringworm starts as a fungal lesion. The fungus often transmits when people share towels or razors.

Ringworm thrives in moist environments, and therefore a person should take care to dry thoroughly after a wash and try not to stay in damp clothes.

Common ringworm symptoms include:

A doctor will prescribe an antifungal treatment to address ringworm.

The best way to prevent ringworm is to wear breathable fabrics, avoid sharing towels and razors, and dry thoroughly after bathing.

Learn more about swollen lymph nodes in the groin here.

Lymphoma is a type of cancer that affects the lymphatic system. The two main types of lymphoma are Hodgkin lymphoma and non-Hodgkin lymphoma.

Hodgkin lymphoma occurs when cancer cells spread from one cluster of lymph nodes to another. By contrast, in non-Hodgkin lymphoma, there is no order in how cancer cells spread throughout the lymphatic system.

Typical symptoms of lymphoma include:

These are also common symptoms of viral infections, which can make lymphoma hard to diagnose. However, in people with lymphoma, symptoms tend to persist for longer periods of time.

It is of note that these symptoms do not clearly indicate cancer. If a person experiences any of these, they should contact a doctor to identify the cause of their symptoms.

Treatment options for lymphoma include:

A person should contact a healthcare professional if they are experiencing persistent swelling of lymph nodes.

Swelling usually indicates an infection, and therefore a person should not immediately worry about lymphoma.

After reaching a diagnosis, a doctor will recommend the appropriate course of treatment.

Lymph nodes are a part of the lymphatic system. They filter lymph, which contains pathogens and damaged cells, and send the dead cells to the kidneys and liver.

Lymph node swelling usually results from an infection. In rare cases, however, it may be due to lymphoma.

If a person is concerned about swelling and other symptoms they have, they should contact a doctor.

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Lymph nodes: Purpose, location, and disease warning signs - Medical News Today