A single dose of the experimental gene therapy AMT-180 promotes clinically meaningful blood-clotting activity, independent of factor VIII levels, in mouse and primate models of hemophilia A, a study shows. These preclinical findings were discussed in a presentation, “Towards AAV5-Mediated Gene Therapy for Hemophilia A with a Factor IX…
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A gene therapy being developed by ASC Therapeutics restored levels of the clotting factor missing in hemophilia A in a mouse model of the disease, and further tests in primates and human cells suggest the therapy might also work for people, the company announced. Results from…
Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.
Point-of-care ultrasonography enables improved diagnostic accuracy, and allows for more targeted treatment and better monitoring of therapy response in hemophilia patients with joint complications, a study suggests. The study, a review of published articles, “Point-of-care Ultrasonography in Orthopedic Management of Hemophilia: Multiple Uses of an Effective Tool,” was published…
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Gene Therapy Seen to Produce FVIIa Levels Needed to Control Bleeding in Rat Study of Hemophilia A
A gene therapy-based factor VIIa (FVIIa) given as prophylactic, or preventative, treatment was seen to produce specific factor levels needed to reduce the frequency of spontaneous bleeding episodes in a rat model of hemophilia A. According to scientists, this work may be a first step in developing therapies that…
To help people understand the science behind hemophilia and other bleeding disorders, digital content agency Believe Limited will present its first science fair this fall. Sponsored by commercial gene therapy company Spark Therapeutics, the science fair will be held at the National Hemophilia Foundation’s 71st Bleeding Disorders Conference…
Doctors in Canada are proposing a set of measures to better judge outcomes and the success of programs helping teen-age hemophilia patients transition to adult care, especially for those with more severe disease. They also listed 15 “key elements” of a successful pediatric-to-adult transition program, including eight the doctors saw as feasible…
A combination of Cyklokapron and Feiba — usually avoided due to a perceived risk of blood clots — appears to be safe and effective for treating patients with acquired hemophilia A (AHA), though these early study results need to be confirmed in clinical trials, researchers in Italy say. The study, “…
Recognizing risk factors of thrombosis and carefully monitoring medication doses are crucial for minimizing thrombotic complications in older patients with acquired hemophilia and serious illnesses like cardiovascular disease, a report describing four cases says. The study, “Acquired Haemophilia A. Which is the best therapeutic choice in older adults? Single…
In recognition of World Hemophilia Day (WHD), Catalyst Biosciences is hosting an “Ask Me Anything” (AMA) session on Reddit on April 17. During the Reddit AMA event, which will take place at noon EST Wednesday, clinicians will answer questions from patients and caregivers about bleeding disorders including hemophilia. A…
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