The first patient has been dosed in a Phase 1/2 study of GB221, a next-generation gene therapy for SMA type 1.

In honor of Rare Disease Day, columnist Halsey Blocher shines a light on the caregivers who make life possible for those with SMA.

Medical equipment and technology are crucial for columnist Ari Anderson, who lives with SMA, so malfunctions can quickly ...

Columnist Helen Baldwin recalls the major life changes that preceded her introduction to the SMA community with the birth of ...

Blood vessel damage in the spinal cord may play a role in nerve cell loss in spinal muscular atrophy, a new study suggests.

Columnist Connie Chandler enjoys volunteer work, but her physical limitations from SMA means she often needs a little help ...

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

A mouse study suggests SMA treatment may partly restore organ-specific redox imbalances linked to SMN protein deficiency.

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.

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

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 ...