Please provide your email address to receive an email when new articles are posted on . This is the first study to investigate treosulfan pharmacogenetics in a large uniform cohort of patients with ...

Credit: Shutterstock. Researchers sought to determine whether luspatercept would improve outcomes in patients with nontransfusion-dependent β-thalassemia. The following article features coverage from ...

Sangamo BioSciences ( SGMO) was founded in 1995 ostensibly to commercialize zinc finger nuclease technology. Twenty years have passed since its founding and there hasn't been a single drug approval ...

Starting iron chelation therapy early may prevent growth problems and organ damage from iron overload in children with TDT. Currently approved medications have limitations for use in very young ...

AAPI Nexus: Asian Americans and Pacific Islanders, Policy, Practice and Community, Vol. 4, No. 2, Youth (Summer/Fall 2006), pp. 111-134 (24 pages) Thalassemia is a potentially life-threatening genetic ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for ...

The success of the treosulfan-based conditioning regimen in patients with β-thalassemia undergoing hematopoietic cell transplantation (HCT) is limited due to several complications, such as mixed ...

About PYRUKYND® (mitapivat) U.S. INDICATION PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.

Avanzanite entered an exclusive partnership with Agios in June 2025 to manage PYRUKYND commercialization across Europe European Commission will now review the CHMP's opinion, with the final ...

- Data Presented at ASH; Company to Host Investor Call on December 10 - CAMBRIDGE, Mass. – December 8, 2014 – bluebird bio, Inc. (Nasdaq: BLUE) today announced data from eight subjects treated with ...