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A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…
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A partnership between Emmes and MedGenome seeks to accelerate development of genomics-powered therapies for people with rare diseases, such as hemophilia. The companies plan to combine patient data into custom disease registries to improve research design and help with patient recruitment for clinical trials. In addition, they…
Canadian Blood Services (CBS) has awarded a three-year tender — an exclusive bulk purchase agreement aiming to minimize medication cost — to Takeda Canada for the purchase of Adynovate, an injectable replacement therapy for the prevention and treatment of bleeding episodes in people with hemophilia A. This…
In an unexpected turn, Catalyst Biosciences is discontinuing the clinical development of marzeptacog alfa activated (MarzAA), its experimental under-the-skin therapy for hemophilia A and B patients with inhibitors, which was being evaluated in an international Phase 3 clinical trial. “Based on several factors including a recently updated feasibility assessment,…
When given monthly to people with hemophilia A or B with inhibitors, fitusiran significantly reduced bleeding, and for some, fully eliminated them, according to data from the Phase 3 ATLAS-INH study.
Painful PTSD memories resurfaced recently for columnist Cazandra Campos-MacDonald’s son following years of his enduring sticks with needles.
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Becoming educated and informed about hemophilia is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
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There is no cure for hemophilia yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.
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