Type 3 spinal muscular atrophy (SMA3) is a milder form of this inherited disease. It’s also called juvenile SMA because it affects children. Another name is Kugelberg-Welander syndrome.

Spinraza (nusinersen) treatment helped school-age children with SMA be more independent in tasks involving their arms and ...

And they did a muscle biopsy on me and were told that we have SMA type 3, which is when you walk ... scoliosis which caused us to have to get a spinal fusion. Unfortunately, one of the bad ...

NMD Pharma Receives IND Clearance to Start a Phase II Clinical Trial of NMD670 for the Treatment of Symptoms of Spinal Muscular Atrophy Type 3 The Phase II clinical trial is a randomized ...

today announces that the first patient has been dosed in a Phase II clinical trial with the ClC-1 inhibitor NMD670 in patients impacted by spinal muscular atrophy (SMA). The patient was dosed ...

Evrysdi is a non-invasive, disease-modifying option that can be swallowed whole or dispersed in water for patients with spinal muscular ... SMA type 1 affects approximately one per 10,000 newborns in ...

SMA gene therapy GC101 was found to rescue disease manifestations in mice and to be safe in non-human primates.

In a hopeful step for medicine, a 2-and-a-half-year-old child born with spinal muscular atrophy (SMA ... The most severe form ...

70. The biotech company Scholar Rock reported Monday that its experimental medicine improved motor function in patients with a muscle-wasting disease called spinal muscular atrophy, paving the way ...