Gene therapy has the potential to stop hemophilia in its tracks. But such therapy will likely be costly, and can carry risks that have generated some apprehension among patients who could benefit most from it. That’s the message from hemophilia patient advocates who spoke at the 2018 NORD Rare Diseases…
A gene therapy from uniQure in early testing, called AMT-180, has the the potential to treat all hemophilia A patients, including those with inhibitors, according to the company. Hemophilia A is caused by missing or defective factor VIII (FVIII), a clotting protein. About 30% of patients with severe hemophilia A develop inhibitors, or…
The workshop and production process over, it was curtains up for “Hemophilia: The Musical,” a unique theatrical event starring high school students affected by bleeding disorders. Performed at New World Stages in New York City on Nov. 12, the premier Broadway-style production rounded out a three-day theater and arts…
uniQure is continuing to enroll participants for its global Phase 3 trial (NCT03569891) testing the potential gene therapy AMT-061 in men with severe or moderately severe hemophilia B, according to an update from the company. In addition, uniQure announced that three patients included in a Phase 2b trial (…
Bioverativ will present some of its most recent therapeutic advancements regarding hemophilia and other blood diseases at the 60th Annual Meeting of the American Society of Hematology (ASH) in San Diego, California, Dec. 1-4. The company, which is part of Sanofi, has focused on the development and commercialization of innovative…
Aptevo Therapeutics provided an update on Ixinity, its hemophilia B therapy, that includes developing a larger 3,000 IU vial assay for patient convenience. The company also plans to initiate a Phase 4 clinical trial in a pediatric patients, and to apply for Ixinity’s approval outside of the United States.
With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…
Hemophilia A patients age 12 and older who live in Canada now may receive prophylactic (preventive) treatment with Bayer’s antihemophilic factor Jivi. Health Canada approved Jivi as a treatment to prevent or reduce the frequency of bleeding episodes in patients age 12 or older who have received…
Re-administering a viral vector-based gene therapy for hemophilia A was shown to be safe and to achieve long-term improvement in a dog model of the disease — even after neutralizing antibodies were evident following the initial treatment. The study, “An observational study from long-term AAV re-administration in…
BioMarin Pharmaceutical Inc. and Believe Limited have announced the students chosen to participate in the theatrical production of “Hemophilia: The Musical,” a program that aims to empower the bleeding community through performance while raising public awareness of the connection between arts and wellness. The 25 high school students hail from…
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