Stay informed with the latest updates for the inheritable blood disorders community.

Industry
Octapharma Announces Final Results of Nuwiq® Study
Industry
The phase 3 trial currently includes 134 participants with hemophilia A, all of whom have received a single dose of the gene therapy.
BioMarin Announces Positive Phase 3 Study Data for Valoctocogene…
Industry
FDA Puts Clinical Hold on uniQure's Hope B Trial
FDA Puts Clinical Hold on uniQure's Hope B Trial
Industry
Catalyst Announces New FDA Designation and Phase 3 MarzAA Study Design
Catalyst Announces New FDA Designation and Phase 3 MarzAA Study Design
Industry
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Pfizer Announces First Participant Dosed Phase 3 Study of Marstacimab
Industry
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uniQure Announces Positive Results from Phase 3 Gene Therapy Trial
Industry
Global Dosing Hold in Fitusiran Trials Initiated by Sanofi Genzyme to Investigate New Adverse Events
Global Dosing Hold in Fitusiran Trials Initiated…
Press Releases
With Patient Perspectives in Mind, ICER Evaluates Hemophilia Therapies
With Patient Perspectives in Mind, ICER Evaluates Hemophilia Therapies
Industry
First Patient Dosed in Phase 3 Gene Therapy Trial
First Patient Dosed in Phase 3 Gene Therapy Trial
Industry
CSL Behring States Stimate Will Not Be Resupplied until 2022
CSL Behring States Stimate Will Not Be Resupplied until 2022
Industry
Sanofi Publishes Updates to BIVV001 Trial
Sanofi Publishes Updates to BIVV001 Trial
Industry
BioMarin’s New Clinical Study to Focus on Antibody Development to AAVs
BioMarin’s New Clinical Study to Focus on Antibody Development to AAVs
Industry
Mylan Initiates Voluntary Nationwide Recall of Four Lots of Tranexamic Acid Injection, USP Due to Carton Label Mix-Up
Mylan Initiates Voluntary…
Industry
FDA Recommends Additional Data for BioMarin’s Investigational Gene Therapy
FDA Recommends Additional Data for BioMarin’s Investigational Gene Therapy…
Industry
Novo Nordisk Announces Near Resumption of Concizumab Trials
Novo Nordisk Announces Near Resumption of Concizumab Trials