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Health Canada Priority Review targets an expedited 180-day review period for givinostat, reflecting Italfarmaco’s commitment to Canadian DMD communityNDS is based on Italfarmaco’s Phase 3 EPIDYS trial...

L’examen prioritaire de Santé Canada vise un délai accéléré de 180 jours pour l’examen du givinostat, reflétant l’engagement d’Italfarmaco envers la communauté canadienne touchée par la dystrophie...

Designation is supported by an exploratory analysis from the Phase 1b study showing that some children with SMA previously treated with gene therapy who had suboptimal clinical status experienced...

Agios provides update on Phase 2b trial of tebapivat in lower-risk myelodysplastic syndromes

LifeArc partners with Elpida Therapeutics to advance gene therapies for three ultra-rare childhood diseases: SPG50, CLN7 and CMT4J.

PYRUKYND (mitapivat) approved in the European Union for adults with thalassemia

SERB Pharmaceuticals to acquire development and commercialisation rights to Idefirix® (imlifidase) in Europe and MENA from Hansa Biopharma for €115 million

Agios announces the submission of its sNDA to the FDA for the U.S. accelerated approval of mitapivat in sickle cell disease

Agios announces new data on mitapivat will be featured in oral and poster presentations at EHA 2026 in Stockholm, Sweden, June 11-14, 2026

White Oak, Md., May 11, 2026 (GLOBE NEWSWIRE) -- The U.S. Food and Drug Administration today announced it is soliciting input on efforts with respect to drug repurposing to help address unmet...