Discover the most-read SMA stories of 2025, featuring updates on the gene therapy Zolgensma, next-generation treatments, and ...

Leuprorelin acetate, a medicine approved in the U.S. for prostate cancer, may slow the progression of SMA type SBMA, ...

Columnist Connie Chandler offers a message of courage and hope for those spending this season in a hospital bed.

A new review of studies reveals that incorporating SMA into newborn screening programs yields clinical improvements and ...

This Christmas, columnist Ari Anderson gives thanks that some health complications he experienced post-surgery resolved on ...

Disability life coaching is turning out to be just the balm columnist Brianna Albers needed to recall the importance of community in SMA.

Fatigue in the diaphragm may contribute to breathing issues in people with spinal muscular atrophy, a new study reports.

Cherie Ehlert lives in Vancouver, British Columbia, Canada. She is a disability advocate and created “Charlie's Angels” to raise funds for children's healthcare services and spinal muscular atrophy, ...

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 ...

The gene therapy Zolgensma (onasemnogene abeparvovec-xioi) has been newly approved to treat patients with spinal muscular atrophy (SMA) through intravenous (IV) delivery, meaning systemically. However ...

SMA news, research and stories delivered to your inbox daily!

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, ...