Columnist Connie Chandler's school days were unremarkable, thanks to the kindness of friends and the special accommodations ...

Children with SMA already experiencing symptoms showed gains in motor abilities after receiving gene therapy, a real-world ...

Researchers at Cedars-Sinai are working on a potential stem cell treatment for three siblings in California with an ultra ...

Columnist Ari Anderson has learned to use his time wisely so that he can enjoy fun activities while also completing the work ...

Keynote speaker Mindy Henderson redefines "impossible" ahead of MDA Engage: Chicago, taking place Sept. 25-26 at the Westin ...

A European survey found that while treatment options have expanded, SMA patients still face limited access to medicines and ...

Columnist Kevin Schaefer celebrates the approval of Isembyld for SMA, and remembers the relentless enthusiasm of his friend ...

The muscle-strengthening agent apitegromab, now Isembyld, has been approved in the U.S. as an add-on treatment for spinal muscular atrophy.

Columnist Brianna Albers used to trust her doctors completely, but having a rare disease forced her to recognize they don't know everything.

Children and adults with SMA and their caregivers may experience clinically relevant mental health symptoms, a German study found.