Spinraza treatment led to sustained improvements in motor function in adults with SMA, according to a South Korean study.

A new study found Spinraza treatment restores a key amino acid called taurine in SMA type 1, which may improve clinical ...

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 ...

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

Today is Caregiver Appreciation Day and columnist Connie Chandler wants the caregivers in her life to know how much she ...

Scholar Rock plans to resubmit its application seeking approval for apitegromab, an investigational muscle-strengthening ...

For columnist Sherry Toh, the new TV series "The Beauty" dwells too much on the grief of living with a rare disease but ...

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

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

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