Children with SMA often struggle to get a good night's sleep, and their caregivers also report feeling sleep-deprived, a new ...

The blood creatinine-to-cystatin-c ratio may be a biomarker of Spinraza treatment response in children with SMA, a study in ...

Guest writer Ronna Johnson feels privileged to support her son, Brandon, in living his best life, pursuing his goals, and ...

Early treatment helps with swallowing and eating for SMA kids, but new research is needed on the effects of disease-modifying ...

Columnist Connie Chandler is inspired by the Olympic athletes, who invite people all over the world to dream that anything is possible.

Treatment with Evrysdi (risdiplam) may modestly improve arm function for adults with spinal muscular atrophy (SMA), a new study indicates.

The strength of nerve electrical impulses in children with SMA can help predict their response to treatment with Spinraza, a study found.

Columnist Sherry Toh writes of her love for Disney's "Beauty and the Beast" and reimagines a production led by a person with a disability.

At first, columnist Connie Chandler wasn't sold on the idea of attending a global prom. But she was soon convinced it's a special night.

Share this page with email Share this page on Facebook Share this page on X Share this page on Reddit Such distinction is based on early observations in animal models of the disease and in people. The ...

After a “constructive and collaborative in-person” meeting with the U.S. Food and Drug Administration (FDA), Scholar Rock is now planning to once again seek approval of its muscle-strengthening agent ...

It’s been almost six years since the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was first approved in the U.S. to treat young children with spinal muscular atrophy (SMA). Now, ...