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This follows confirmation that no appeals were received against the Final Draft Guidance (FDG) recommendation announced on December 10, 2024.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), ...

The Haslam family knew early on that their 10-year-old and 12-year-old were suffering from muscular dystrophy, but what they didn't know is that one day, the University of New Mexico Hospital would ...

Stephanie Leavins was born with rare form of Muscular Dystrophy, and her parents were urged to abandon hope of even bringing her home. For 47 years she inspired everyone around her.

who has spent his life helping sick children. On redirect, I asked him what he does with his royalties, and he explained that he sets some aside for his sick wife (who, tragically, has a presently ...

The latest addition to its lineup, the Paediatric Robotic Gait Trainer, is specifically designed for the rehabilitation of children with conditions such as cerebral palsy, brain injuries, muscular ...