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

Industry
uniQure Announces Findings from HCC Case Investigation in HOPE-B Trial for Gene Therapy
uniQure Announces Findings from HCC Case Investigation
Industry
Ampoule bottles on an assembly line
Update to Hemlibra® (emicizumab-kxwh) USPI
Industry
New Program Looks to Standardize Care for Patients with Rare Diseases
New Program Looks to Standardize Care for Patients with Rare Diseases
Industry
FDA Partially Rescinds Added Indications for Two FIX Products
Industry
Blood Cells Image
Sanofi Revises Fitusiran Dosing Regimen to Mitigate Risk of Vascular…
Industry
Spark Announces Preliminary Data from Investigational Gene Therapy Trial
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
1174384721-doses
Pfizer Announces First Participant Dosed Phase 3 Study of Marstacimab
Industry
1242454551-Medical-Trial
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