Defeat Duchenne Canada is proud to celebrate its 30th anniversary, marking three decades of relentless progress in the fight ...

Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will host its sixth in-person Duchenne Healthcare Professionals ...

Initiation of Phase 1b study using SGT-212 for the treatment of patients with Friedreich's Ataxia expected in 2H25. Click ...

Santhera Pharmaceuticals’ Agamree (vamorolone) has been accepted by the Scottish Medicines Consortium (SMC) to treat Duchenne ...

Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

Analyst Michael Ulz of Morgan Stanley maintained a Buy rating on Dyne Therapeutics (DYN – Research Report), retaining the price target of ...

Santhera has begun launch preparations for AGAMREE® in UK with first sales expected in this quarterPratteln, Switzerland, January 16, 2025 – ...

Vamorolone (Agamree) is available on the NHS as a possible treatment for Duchenne muscular dystrophy (DMD) in people 4 years and over. Is this treatment right for me? Your healthcare professionals ...

Santhera Pharmaceuticals will launch its new drug for Duchenne muscular dystrophy in the UK within the next few weeks, after ...

Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...

A trial team led by Latham’s Mike Morin and David Frazier and Finnegan’s William (Bill) Raich helped Sarepta Therapeutics ...