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 ...

Insmed is set to challenge the Duchenne muscular dystrophy (DMD) treatment space with its innovative gene therapy asset, INS1201.

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 ...

To read further articles in this series please visit our Disease Spotlight: Duchenne Muscular Dystrophy (DMD) channel produced by pharmaphorum in conjunction with Santhera Pharmaceuticals.

Capricor's biologics license application for deramiocel (CAP-1002) is draws largely on the Phase 2 HOPE-2 trial and its ...

Capricor Therapeutics has completed its US marketing application for deramiocel, a cell therapy the US biotech hopes will ...

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, ...

As of November 2024, approximately 156 clinical trials for Duchenne muscular dystrophy (DMD) are ongoing or pending ... (including 7 patient registries), and 106 are exploring potential interventions ...

This year’s top stories on Duchenne muscular dystrophy (DMD) managed care included a novel imaging method for tracking disease progression, an FDA approval of the first nonsteroidal therapy ...