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

Industry
Spark Announces Multiyear Data Update for Investigational Gene Therapy
Spark Announces Multiyear Data Update for Investigational Gene Therapy
NBDF & Community
Washington D.C. - Capitol Building
NHF Applauds Implementation of the Hemophilia SNF Access Act
Medical
Prophylaxis Indication Restored for BeneFIX®
Industry
Spark Provides Trial Updates for Investigational Gene Therapy SPK-8011
Spark Provides Trial Updates for Investigational Gene Therapy SPK-8011
Industry
FDA Places Clinical Hold on Sigilon’s Investigational Hemophilia Therapy
FDA Places Clinical Hold on Sigilon’s Investigational Hemophilia Therapy
Industry
Female scientist conducting research in lab with goggles and gloves
BioMarin Presents Positive Safety and Efficacy Data for their…
Industry
Gene Therapy Trial Data Suggests Efficacy in the Presence of Antibodies
Gene Therapy Trial Data Suggests Efficacy in the Presence of Antibodies
Industry
Gene Therapy Licensing Agreement Between uniQure and CSL Now in Effect
Gene Therapy Licensing Agreement Between uniQure and CSL Now in Effect
Industry
Gene Therapy Helix
uniQure Announces End of Clinical Hold on Hemophilia B Gene Therapy…
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