Novartis has confirmed that at least two reports of tumors appear to have been caused by treatment with the gene therapy ...

While columnist Alyssa Silva has a lot of experience with certain health procedures, that doesn't make them easier to ...

SMA treatment with Spinraza was linked to early motor improvements and long-term stability in people with SMA types 2 and 3, ...

I hadn’t even used AI for research until a couple months ago. Moreover, the ease with which you could generate 50,000 words ...

Spinraza improved motor function in children and adolescents with SMA in a real-world study from Czechia and Slovakia.

Two interconnected themes — community and hope — dominated conversations at this year's MDA Engage symposium in Dallas.

Columnist Halsey Blocher was impressed by the accessibility and inclusivity she encountered in a small Bavarian town in Michigan.

As part of his ongoing efforts to encourage others, columnist Ari Anderson shares his plans to write a book about his life ...

Melatonin and pioglitazone, two approved drugs that act on metabolism, improved motor function and prolonged survival in ...

Canada’s Drug Agency recommends expanding public funding for Biogen’s Spinraza to include adults and more patients with SMA.

What is SMA type 2? Spinal muscular atrophy (SMA) type 2 is a rare genetic disease also known as Dubowitz disease or intermediate SMA. It is characterized by muscle weakness and wasting. This form is ...