News

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…

SMA adults recommend tailored patient-reported outcome measures

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…

DMTs may reduce risk of children’s bone fractures in SMA: Study

Disease-modifying therapies (DMTs) may help lower the risk of children’s bone fractures among young patients with spinal muscular atrophy (SMA), a study by U.S. researchers suggests. “Drug therapy led to a decrease in fracture occurrence,” the scientists wrote, noting that “patients on treatment had fewer fractures compared [with] pretreatment.”…

MDA Clinical & Scientific Conference starts on March 16

Note: This story has been updated March 6, 2025, to correct a quote from Mindy Henderson, MDA vice president of disability outreach and empowerment. The Muscular Dystrophy Association (MDA) is once again hosting its annual gathering, the MDA Clinical & Scientific Conference. The 2025 event will take place…

Mitochondria is a potential target for SMA treatment: Study

The parts of cells that produce energy, called mitochondria, in the skeletal muscles involved in voluntary movements don’t work properly in spinal muscular atrophy (SMA) and may contribute to the disease’s symptoms, a study in mice and cells suggests. Engaging mice in a single session of exercise or adding…