UniQure N.V. has announced that is has “scaled up” the manufacturing process at its large facility in Lexington, MA.
The collaboration between stakeholders to establish a core set of measurements that would be used to evaluate effectiveness of gene therapies.
BioMarin Pharmaceutical, Inc. recently provided updated interim results from a phase 1/2 clinical study of BMN 270, the company’s investigational hemophilia A gene therapy.
The Committee of Ten Thousand has chosen not to participate in nor support any fundraising efforts for the hemophilia memorial project.
The program will allow bleeding disorders camp staff learn from one another and share best practices.
This activity aims to enable the learner to apply knowledge of the benefits and applications of paper and electronic infusion logs.
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.
NHF, HFA, the Hemophilia Alliance and Coalition for Hemophilia B are united in their opposition to the Graham-Cassidy-Heller-Johnson Bill because of its damaging impact on our community and urge the Senate to reject these harmful policies.
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