For people living with neuromuscular diseases such as spinal muscular atrophy (SMA), listening to the body and recognizing fatigue and pain is only half the battle. The other half is communicating that information to their care team so they can help. “Pain is a subjective experience,” said Jenny Riecke,…
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Treatment with Spinraza (nusinersen) led to sustained improvements in motor function in children and adolescents with spinal muscular atrophy (SMA), according to a real-world study from Czechia and Slovakia. Improvements were generally greatest during the first months of therapy and more pronounced in younger children, with benefits largely…
Treatment with melatonin and pioglitazone, two approved drugs that act on metabolism, improved motor function and prolonged survival in a mouse model of spinal muscular atrophy (SMA), with melatonin having the greater impact on survival, a study showed. The findings provide “strong rationale for exploring metabolism-targeted second-generation therapies to…
Treating adults with late-onset spinal muscular atrophy (SMA) with Evrysdi (risdiplam) partially normalized changes in certain RNA molecules found in blood, a study found. These molecules — cell-free messenger RNA (ccfmRNA) — are associated with inflammation and cellular stress. They are found in various bodily fluids, including blood,…
Treatment with Evrysdi (risdiplam) was associated with stable overall motor function and improved upper limb function in a small Chinese study of 15 people with spinal muscular atrophy (SMA). Upper limb function scores increased in the type 2 group and the combined types 3–4 group, although neither change…
This Halloween, an 11-year-old living with spinal muscular atrophy (SMA) named Dash will be trick-or-treating in a costume fit for a galaxy far, far away. Last year, Dash Stanton was selected as the winner of a Halloween sweepstakes on Instagram that was run by the Muscular Dystrophy Association (MDA)…
Lily Sander will never forget what it was like to meet other people with neuromuscular diseases the first time she attended Muscular Dystrophy Association (MDA) Summer Camp. Living with Charcot-Marie-Tooth disease, Sander had met only one or two other people with neuromuscular diseases in her city. She was…
Health-related quality of life for spinal muscular atrophy (SMA) patients treated with Spinraza (nusinersen) is closely linked to the well-being of their caregivers, particularly among patients who are able to sit, a South Korean study found. Participants with SMA who had poorer motor function generally reported lower quality…
Scholar Rock is working to bring spinal muscular atrophy (SMA) treatment Isembyld (apitegromab-mstn) to patients worldwide, following U.S. Food and Drug Administration (FDA) approval of the infusion therapy as the first muscle-strengthening agent to treat the disease. “Our work does not stop with the US approval,” Akshay…
After its success in the U.S., Scholar Rock is once again seeking approval in the European Union for its muscle-strengthening drug apitegromab —now launching as Isembyld in America — for spinal muscular atrophy (SMA). The developer has resubmitted an application to the European Medicines Agency (EMA)…
