News

MDA 2025: Spinraza treatment stabilizes adult motor function

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…

MDA 2025: Potassium channel could be SMA treatment target

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…

MDA 2025: High-dose Spinraza safe, effective in DEVOTE study

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…

MDA 2025: Apitegromab safely improves SMA motor function

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…

MDA 2025: Evrysdi consistent after 5-year SUNFISH follow-up

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…

MDA 2025: SMA kids maintaining Zolgensma benefits 10 years later

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…

MDA 2025: SMA gene therapy OAV101 shows acceptable safety

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…

MDA 2025: Conference begins, marking another year of progress

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…

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