Mononine, a treatment for hemophilia B, hit the market nearly three decades ago in 1992. Since then, there have been amazing advancements in the knowledge and treatment for this disease.…
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…
As society becomes increasingly technological, researchers are getting closer and closer to mapping the entire human genome. In facts, shares Medical XPress, we may be nearly there. While many…
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…
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…
According to a release on Public News Service, the state of West Virginia just implemented a new bipartisan law to support individuals with rare diseases and their families, a particular…
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…
A recent article published in India’s Pharma Literati cites The World Health Organization as not having endorsed a definition for rare diseases. The European Union defines 'rare' as less than…
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…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…
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,…
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