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…
News
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…
OAV101 IT, a version of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s administered into the spinal canal, can be safely given to people with spinal muscular atrophy (SMA) who were previously treated with other SMA therapies, according to new data from a clinical trial. “No [adverse events] leading…
The Muscular Dystrophy Association‘s (MDA) annual Clinical & Scientific Conference kicked off over the weekend, bringing together patients, caregivers, researchers, clinicians, academics, advocates, and industry leaders to discuss the latest in science and care related to a range of neuromuscular diseases. This year’s conference will bring together hundreds…
Adults with spinal muscular atrophy (SMA) recommend individually tailored patient-reported outcome measures, or PROMs, to accurately gauge their lived experience, with a focus that goes beyond assessments of physical function to incorporate mental health, sexual function, sleep, and fatigue. “New or modified patient-reported outcome measures should be developed collaboratively…
