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.…
According to a story from biopharmadive.com, the field of gene therapy has gained increasing relevance throughout 2019, and 2020 will likely be a continuation of this trend. An area of…
In a story for Hemophilia News Today, author Shellye Horowitz has written a piece titled "An Open Letter to Providers Treating Women with Hemophilia." Women still face discrimination and unequal…
According to a story from BioPortfolio, the drug company Octapharma USA sponsored the 71st Bleeding Disorder Conference, which is put on by the National Hemophilia Foundation. The company is also…
According to a story from Hemophilia News Today, the drug developer uniQure has completed its goals for patient enrollment ahead of time for is phase 3 clinical trial. This clinical…
According to a story from MD Edge, a phase 2 clinical trial testing the drug concizumab as a treatment for hemophilia A and hemophilia B demonstrated substantial capability in reducing…
According to a publication from Hemophilia News Today, an international team of researchers with members from France and the United Kingdom recently conducted a study of over 100 hemophilia patients…
Camp Notaclotamongus In Eureka, Missouri, Camp Notaclotamongus has some typical activities for a summer camp. They state that camper goals aim to build friendships, confidence, and personal growth with outdoor…
Blood Donations For many people living with a rare disease such as Beta thalassemia, Hemophilia, Aplastic Anemia, Myelodysplastic syndromes, Acute Promyelocytic leukemia, and others, blood transfusions are not uncommon. For…
According to a publication from Reuters, based on early clinical data, American biotechnology company BioMarin Pharmaceuticals believes that a single injection of its experimental hemophilia A drug could relieve symptoms…
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