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

A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...

A new drug-free, minimally invasive intervention targets the root cause of progressive loss of neural function in spinal muscle atrophy (SMA), an inherited neuromuscular disease.

is confident of bringing a change in the lives of countless children, like her, who are living with Spinal Muscular Atrophy (SMA) with the support of her mother, sister and teacher. Ms. Keya was ...

The primary endpoint of Part B was the change from baseline on The Children’s Hospital of Philadelphia ... and-ema-accept-applications-higher-dose-regimen-nusinersen 2. Spinal Muscular Atrophy: ...

Seeking urgent Union government intervention to overcome the lack of affordable access to medicines for treating Spinal Muscular Atrophy (SMA ... are nearly 8,000 children to 25,000 children ...

SPINRAZA is an approved treatment for spinal muscular atrophy in over 71 countries ... open-label evaluation of the higher dose regimen in children and adults who transitioned from SPINRAZA ...

Kate crouched down to chat with the children with life-shortening ... aged three, who has spinal muscular atrophy and was dressed in pink and sat in a matching wheelchair. The future Queen dropped ...

Patients with spinal muscular atrophy (SMA) type 2 showed improved ... pediatric neurologist and a principal investigator at Children's Hospital of the King's Daughters, said in a Novartis news ...