The Muscular Dystrophy Association (MDA) has selected Lily Sander from Charlotte, North Carolina, as its 2025 MDA National ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

As a parent and caregiver to three sons with DMD, columnist Betty Vertin's definition of what success looks like has changed over the years.

The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to NS-051/NCNP-04, which is ...

We are pleased to announce that our applications for the higher dose regimen of nusinersen are now under review in the US and Europe,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular ...

Behind every photograph lies a story, and for Alexander Amora Juni, it's a story of love, sacrifice, and unwavering ...

Use precise geolocation data and actively scan device characteristics for identification. This is done to store and access ...

This follows confirmation that no appeals were received against the Final Draft Guidance (FDG) recommendation announced on December 10, 2024. Following this, Santhera has already started launch ...

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

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 ...

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