Adults with SBMA have elevated levels of NfL and GFAP, two biomarkers for nerve damage, in their cerebrospinal fluid, a study ...

As he celebrates SMA Awareness Month, columnist Ari Anderson reflects on his desire to have fun — on day trips as well as ...

Columnist Sherry Toh realizes — through dating apps! — just how much she lives a life of privilege even as she's living with ...

A decision on apitegromab is due by Sept. 22, with Scholar Rock planning to make it available in the U.S. this year, if it is ...

Wow, does that resonate! This disability and all the physical and emotional struggles that come with it is not the life, the ...

Zolgensma is approved for any type of SMA related to SMN1 mutations, but shouldn't be administered to anyone with an active ...

Impressed by the accurate portrayal of emergency rooms on "The Pitt," columnist Sherry Toh hopes to see her own rare disease ...

Columnist Connie Chandler explains how letting go of her "ideal" checklist helped her build a diverse and reliable team of ...

SMA Awareness Month is a time to shine a light on the everyday challenges and celebrations with this disease, writes columnist.

Columnist Helen Baldwin observes SMA Awareness Month by recalling her son's short life with the disease, as well as all she ...

A man with SMA and no history of diabetes developed diabetic ketoacidosis, showing how muscle loss may disrupt metabolism.

The nerve cell-targeting EXG001-307 seems to have a better safety profile than Zolgensma, showing fewer adverse effects on ...