Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...

Currently, the DMD treatment landscape includes Sarepta’s Elevidys (delandistrogene moxeparvovec), the first FDA-approved ...

When Raniya Scott was just two years old, her world changed with an unexpected diagnosis: Duchenne muscular dystrophy (DMD), ...

Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic ...

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

Santhera gets positive recommendation from Scottish Medicines Consortium for use of Agamree to treat Duchenne muscular dystrophy: Pratteln, Switzerland Wednesday, January 15, 2025 ...

If you want to get healthy in the new year, it’s important to focus on all body parts, including your mouth. Anne Clemons, ...

GIVI-MPC, a stem cell therapy for certain types of muscular dystrophy, has been granted orphan drug status for treating ...

Sarepta's current exon-skipping DMD therapies – Exondys 51 (eteplirsen ... Reimagining What's Possibl In a new white paper from the World Without Disease initiative, a 2024 update is provided ...

As the year gets underway, analysts and biotech executives highlight cell therapy’s pivot from oncology to autoimmune ...

There have been few heartwarming storylines in a year full of struggles for the New York Jets. A team that had Super Bowl ...