Latest News and Press Releases

NEW YORK, June 22, 2026 (GLOBE NEWSWIRE) -- CurePSP announced the latest recipients of the Collaborative Approaches to Resources, Education and Support (CARES) grants, awarding a total of $155,000...

WATERTOWN, Mass. and CINCINNATI, June 18, 2026 (GLOBE NEWSWIRE) -- Constantiam Biosciences and Cincinnati Children's today announced a strategic collaboration through an exclusive option for future...

Pharmaceutical executive brings 25+ years of international commercial leadership and rare disease experience Egham, UK – 18 June 2026 – Essential Pharma ("Essential" or "the Company"), a global...

First patient dosed in FPWR-supported Phase 2a trial of CSTI-500 for hyperphagia and neurobehavioral symptoms in PWS.

Agios showcases RISE UP Phase 3 results at EHA 2026 Plenary Session reinforcing strong anti-hemolytic profile of mitapivat in sickle cell disease

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.