A new study found Spinraza treatment restores a key amino acid called taurine in SMA type 1, which may improve clinical ...
The first patient has been dosed in a Phase 1/2 study of GB221, a next-generation gene therapy for SMA type 1.
Columnist Helen Baldwin recalls the major life changes that preceded her introduction to the SMA community with the birth of ...
Medical equipment and technology are crucial for columnist Ari Anderson, who lives with SMA, so malfunctions can quickly ...
Columnist Connie Chandler enjoys volunteer work, but her physical limitations from SMA means she often needs a little help ...
In honor of Rare Disease Day, columnist Halsey Blocher shines a light on the caregivers who make life possible for those with SMA.
Blood vessel damage in the spinal cord may play a role in nerve cell loss in spinal muscular atrophy, a new study suggests.
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.
Children with SMA often struggle to get a good night's sleep, and their caregivers also report feeling sleep-deprived, a new ...
Connie Chandler is the director of family engagement at the nonprofit organization We Carry Kevan. She grew up in the foothills of North Carolina but now owns a home and lives in Fort Wayne, Indiana.
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