Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal stimulation.

Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.

Tony, from Skellingthorpe, Lincoln, was diagnosed with myofibrillar myositis and anti-SRP immune-mediated necrotizing ...

At just 15 years old, Ragheeb has already made a tremendous impact on his community as an ambassador for Capes for Kids, an ...

Four siblings came together to create hilarious 50th wedding anniversary present for their parents — re-creating their iconic ...

Tribune News NetworkDohaSidra Medicine, a member of Qatar Foundation, has established a Gene Therapy Center to treat rare genetic diseases such as Spinal Muscular Atrophy (SMA) and Duchenne Muscular ...

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

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 ...

Your support will help my child's weekly medication (P3,300) for Duchenne Muscular Dystrophy. Your recommendation would ... his passion for photography with the challenges of raising his children, ...