Last month, biotechnology company BioMarin sought approval for an investigational gene therapy (valoctocogene roxaparvovec) for patients with hemophilia A. However, their approval was denied. Instead, the company was told that…
According to a story from hemophilia.org, the drug company Ferring Pharmaceuticals issued a recall of the nasal spray drug desmopressin (marketed as Stimate) on July 21st, 2020. This product is…
Last Tuesday, the FDA rejected BioMarin Pharmaceutical's gene therapy, Roctavian, for patients with hemophilia A. According to ABC News, the gene therapy infusion was meant to be a single treatment…
During the 2020 Virtual Congress of the International Society on Thrombosis and Haemostasis (ISTH 2020), which took place from July 12-14, participants discussed the latest in treatments, research, and…
Happy Friday! We're more than halfway through July. Where is the summer going? As a reminder, it's Juvenile Idiopathic Arthritis Awareness Month and if you're looking for ways that you…
According to a story from theindychannel.com, brothers Elias, age eleven, and Emmett, age seven, were both born with the rare bleeding disorder hemophilia A. However, this has not stopped them…
At the end of June, the National Hemophilia Foundation (NHF) released a warning to patients with hemophilia regarding AT132, an investigational gene therapy candidate. Developed by Audentes Therapeutics, AT132 uses…
By Lauren Thayer from In The Cloud Copy Hemophilia is a medical condition in which a person’s blood clotting ability is severely reduced. This inability to properly clot leads to…
The World Federation of Hemophilia's Virtual Summit: Connecting the Global Bleeding Disorders Community June 15-19, 2020 In light of the cancellation of WFH's World Summit due to the ongoing coronavirus/COVID-19…
By Danielle Bradshaw from In The Cloud Copy The cells of hemophilia A patients were taken and genetically modified so that they would create an active clotting factor VIII (or…
The medical realm is changing - but how does that impact patient care and clinical expectations? The National Hemophilia Foundation discusses how clinical expectations for patients with hemophilia don't necessarily…
According to Hemophilia News Today, data collected from a Phase 2b trial highlights that the subcutaneously administered DalcA is effective for the treatment of hemophilia B. Find the full press…
Are opioids an effective and frequently-prescribed treatment for hemophilia? Actually, according to Hemophilia News Today, opioids are prescribed at a much higher rate than reported. This suggests that doctors and…
Patients who better understand their condition and the necessity of treatment are more likely to experience positive outcomes, says Hemophilia News Today. This is because increased awareness and education correlates…
When Shellye Horowitz was younger, she had a clear link to the rare disease community: her father. He had hemophilia and, much like many patients, relied on blood clotting factor…
Patients with hemophilia may experience sudden bleeding, joint pain and inflammation, and deep bruising. But according to Hemophilia News, two proteins may be the cause. Researchers found that patients…
Recently, Pfizer's Dr. Ian Winburn sat down with Europabio for an interview in honor of World Hemophilia Day, which just occurred on April 17. In his discussion, Dr. Winburn…
April 17th, 2020 is World Hemophilia Day. This event is held to help foster awareness about hemophilia and other related bleeding disorders. It is a day when this rare community…
Rare disease advocates in India are pushing for the Health Ministry to create a new fund for rare diseases. Additionally, they're pushing for medicines and medical equipment used for these…
The Institute for Gene Therapies (IGT) is a new development, aimed at improving the speed of development and accessibility of gene therapies. Gene Therapy Many rare diseases are caused by…
AskBio Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina. This company is dedicated to the research of gene therapies as potential therapeutic options…
According to a story from newsroom.heart.org, one of the most substantial domestic policy achievements of the Obama administration was the passage of the Affordable Care Act (ACA). Since its passage,…
Hemophilia A is the most common type of hemophilia, accounting for 8 of every 10 cases. Treatment in the past consisted of the administration of concentrated factor VIII through…
According to a story from biopharmadive.com, the race to a hemophilia A gene therapy continues to tighten following a recent release of early data from Sangamo Therapeutics and Pfizer. The…
By Caitlin Seida from In The Cloud Copy People living with the severe hemophilia A may soon see a new treatment option available thanks to the work of BioMarin Pharmaceuticals.…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
