The program will allow bleeding disorders camp staff learn from one another and share best practices.
Alnylam reports the latest Phase 2 study showed that the safety and tolerability associated with fitusiran “remains encouraging.”
If effective, the therapy prompts the sustained production of FVIII and enhanced protection from bleeds.
Results showed a statistically significant and clinically meaningful reduction in bleed rate, reports states.
FDA approves the Biologics License Application (BLA) for REBINYN®
New therapy failed to show significant efficacy in a phase I/II clinical study.
An interim analysis after a median of 12 weeks of prophylaxis with emicizumab showed a “clinically meaningful” reduction in the number of bleeds over time.
The company will donate a minimum of 140 million international units (IU) of factor VIII to the WFH Humanitarian Aid Program over the next five years.
Spark reports that all participants have experienced “consistent and sustained increases in factor IX activity” following administration of SPK-9001.
Shire has announced a phase 3 clinical trial for ADYNOVATE®, the company’s recombinant factor VIII (FVIII) therapy designed using pegylation technology to extend FVIII time in circulation and increase FVIII exposure to enhance bleed prevention.
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