Using wearable sensors during clinical tests may help doctors objectively measure changes in mobility in people with SBMA, a ...

Combining genetic tests may better define complex SMN gene changes in SMA, including SMN2 copy numbers and hybrid structures.

Columnist Jasmine Ramos has found dating with a disability to be frustrating and emotionally exhausting, but she holds on to ...

Salanersen, a new Biogen treatment for SMA, was awarded breakthrough therapy status by the U.S. Food and Drug Administration. This potent therapy, similar to Spinraza, requires fewer doses (once ...

This article was provided by our partner, the Muscular Dystrophy Association. It has been reviewed by Bionews for accuracy ...

Extracellular vesicles (EVs) are potential biomarkers for SMA progression and Spinraza treatment response. Untreated SMA patients show elevated EV levels, which decrease with Spinraza therapy.

Researchers used long-read sequencing to find two previously unknown SMN1 deletions in two women with mild adult-onset SMA.

In episode 158, host Kevin Schaefer talks with Alberto Lopez from Los Angeles County, California. Alberto is a consultant and… ...

Novartis has completed negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) regarding pricing and availability of Zolgensma (onasemnogene abeparvovec), its approved gene therapy for ...

A few weeks ago, I saw the award-winning musical “Wicked” with my sister-in-law. I saw the show back in 2012 with my parents in New York City, and I jumped at the chance to buy tickets when I saw it ...

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, ...