The Independent Data Monitoring Committee recommended transitioning patients in the placebo arm to garetosmab as soon as possible. Topline data were announced from a phase 3 trial evaluating ...

BELLMAWR, New Jersey (WPVI) -- A young South Jersey boy has one wish for his birthday this year, and that is to help raise money for those living with a rare disease. Nine-year-old AJ Gonzales of ...

PARIS, FRANCE, 19 December 2025 - Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the pivotal Phase II FALKON trial did not meet its primary endpoint of reducing new heterotopic ossification ...

Palovarotene is the first medicine to be submitted for regulatory approval for fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease with an estimated prevalence of 1.36 per million ...

FOP is a disease in which muscles, tendons and ligaments are progressively replaced by bone, leading to eventual incapacitation Garetosmab is the first and only treatment to demonstrate a dramatic ...

The CRL is related to the U.S. FDA’s previous request for additional information on palovarotene clinical trial data Ipsen anticipates responding to the request in the first quarter of 2023 “Although ...