News

The balance of amino acids capable of influencing neurological function is disrupted in patients with spinal muscular atrophy (SMA) and in mouse models, a study found. Observed changes in patients were partially normalized after treatment with the disease-modifying therapy Spinraza (nusinersen). Treatment with one of the amino acids,…

A tablet formulation of Evrysdi (risdiplam) for spinal muscular atrophy (SMA) recently approved in the U.S. is designed to give patients a new option, a Genentech official said. “Evrysdi is the only oral, non-invasive disease-modifying SMA treatment, and the new tablet formulation may provide greater freedom and…

Treatment with Spinraza (nusinersen) can help stabilize motor function in adults with spinal muscular atrophy (SMA), according to a new analysis. “This study aligns with previously reported findings and suggests that long-term [treatment with Spinraza] offers sustained clinical benefits in adults with SMA,” researchers wrote in the abstract…

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, more than 95% of babies diagnosed with SMA are being treated with Zolgensma, according to Daniel Grant, vice president…

The muscle-strengthening therapy apitegromab is being considered for approval by the U.S. Food and Drug Administration (FDA) as an add-on treatment for spinal muscular atrophy (SMA), with a decision expected around the start of fall. Scholar Rock, the company developing apitegromab, applied seeking FDA…

Activating a protein called potassium channel Kv2.1 may help improve motor function in spinal muscular atrophy (SMA), according to data from experiments done in mice that point to the protein’s potential as a target in SMA treatment. The findings were presented at the Muscular Dystrophy Association‘s 2025 MDA…

A higher dose of Spinraza (nusinersen) may be more effective than the currently approved dosing schedule in people with spinal muscular atrophy (SMA) for maintaining motor function, according to data from the DEVOTE clinical trial. Results from the study, which was sponsored by Spinraza’s maker Biogen, were…

Apitegromab, a muscle-strengthening therapy up for approval in the U.S., led to gains in motor function in a clinical trial for people with spinal muscular atrophy (SMA) who took disease-modifying therapies. Data from the trial, called SAPPHIRE (NCT05156320), formed the basis of Scholar Rock‘s recent…

After five years of treatment with Evrysdi (risdiplam), people with spinal muscular atrophy (SMA) types 2 and 3 continue to see stabilization and improvement in measures of motor function. That’s according to new long-term data from the Phase 2/3 clinical trial SUNFISH (NCT02908685), sponsored by Roche, which…

Children with spinal muscular atrophy (SMA) given the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in infancy are maintaining motor milestones after up to a decade of follow-up, according to new long-term clinical trial data. “At their last assessment, … patients [given the now-approved dosage of Zolgensma] were able to…