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

Industry
BioMarin Provides Update Including Next Steps for Investigational Gene Therapy
Trial Data for Roche Investigational SubQ Hemophilia Therapy Presented…
Industry
Gene Therapy Helix
Integration of Spark into Roche Group Set, Hemophilia Gene…
Industry
Doctor and woman looking at computer screen in doctors office
First Patient Dosed with Investigational Sub-Q Therapy for von…
Industry
Lab work and technician
Pfizer Discontinues Beqvez Hemophilia B Gene Therapy
Industry
Red blood cells bone marrow
Investigational Sub-Q Von Willebrand Disease Therapy Put on Fast Track
Industry
DNA structure
Trial Updates for Investigational Hemophilia A Gene Therapy Shared at ASH…
Industry
A person pointing at their arm with purple spots
Sanofi Announces Positive Data for Its Investigational ITP Therapy
Industry
A subject matter expert giving a lexure to a group
Star Therapeutics Presents Data on Investigational VWD Therapy
Industry
Person reading the paper
BioMarin to Narrow Its Commercial Focus for Hemophilia A Gene Therapy
Industry
Gene Therapy a pill with DNA
Pfizer Announces New Data from Hemophilia A Gene Therapy Study
Industry
FDA Approval Includes Full ALTUVIIIO™ Pediatric Trial Results
Industry
Gene Therapy
Hemophilia B Gene Therapy Trial Data Published
Industry
Persons hand with a rash
Takeda Announces Interim Trial Results for Investigational ITP Therapy
Industry
uniQure Announces Findings from HCC Case Investigation in HOPE-B Trial for Gene Therapy
Takeda's Voluntary Withdrawal: Alert on Misprinted Labels for VONVENDI®
Industry
FDA Accepts Biologics License Application for Marstacimab