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

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

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

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

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

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

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 ...

Spinal muscular atrophy, or SMA, is a genetic disorder that is divided into five main types. Historically, people were categorized based on the age at which symptoms first appear, but now, in the ...

Spinal muscular atrophy (SMA) is a rare genetic condition characterized by progressive muscle weakness and atrophy. It mainly affects motor function, but often also causes problems with speaking, ...

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 ...

What is Evrysdi for SMA? Evrysdi (risdiplam) is a disease-modifying therapy, taken daily, that’s approved for children and adults with spinal muscular atrophy (SMA). It is the only approved SMA ...