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.
Search results for:
The investigational gene therapy SB-525Â is tolerated well by patients, and dose-dependent responses are positive, according to preliminary results of a Phase 1/2 clinical trial of hemophilia A. The potential gene therapy is being developed as a partnership between Sangamo Therapeutics and Pfizer. It consists of…
Despite the COVID-19Â pandemic, BioMarin Pharmaceutical‘s application to the U.S. Food and Drug Administration (FDA) seeking approval of valoctocogene roxaparvovec, an experimental gene therapy for adults with severe hemophilia A, remains on track. During a conference call, the company also said that if approved, the…
A web-based software to help personalize dosing regimens for some hemophilia A patients has received marketing clearance from the U.S. Food and Drug Administration (FDA). The 510(k) marketing clearance was granted to Shire, the developer, following its submission of a “premarket notification,” which is necessary if a new device is…
The association of hemophilia A (AHA) and myelofibrosis, a bone marrow disorder, is uncommon and rarely described but in a recent case study, researchers at the Centre Hospitalier Metropole Savoie, in France, reported a patient with AHA secondary to hematological malignancy whose rapid and challenging diagnosis — and timely treatment — was…
Hemophilia is a rare disorder in which a person’s blood is not able to clot properly, resulting in unusually easy and prolonged bleeding.
Precision BioLogic has developed what it says is a better way of detecting levels of substances in hemophilia A patients’ blood that prevent clotting factor therapies from working as well as they should. It collaborated with Roche and Genentech in creating a kit it says can detect levels of the…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to marzeptacog alfa activated (MarzAA), a lab-made version of clotting factor VIIa for the subcutaneous (under-the-skin) treatment of episodic bleeds in hemophilia A and B patients with inhibitors. MarzAA, developed by Catalyst Biosciences, will…
Up to 14 months after a one-time infusion of SB-525, an investigational gene therapy, durable increases in the activity of clotting factor VIII (FVIII) are continuing in all five severe hemophilia AÂ patients treated at the highest dose in the Alta Phase 1/2 trial. None of these…
A generic version of DDAVP (desmopressin acetate), a therapy approved to control bleeding episodes in people with mild hemophilia A, is available in the U.S. Avenacy planned to begin shipping the therapy the week of April 15, according to a company press release. The generic is available…