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

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

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 approved.

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

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

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 ...

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

Columnist Ari Anderson still needs to expand his team of home-care nurses, but the ones he has always go the extra mile.

In honor of SMA Awareness Month, columnist Halsey Blocher is remembering loved ones by trying to live in the same way they did.

The financial costs of SMA include medications, therapy, and assistive devices, and can be offset by health insurance and financial aid.