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…
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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)…
Babies born in maternity wards across the West Midlands, in England, are now being tested for spinal muscular atrophy (SMA) as part of a new National Health Service (NHS) initiative launched on Oct. 1. The regional rollout marks the first phase of a broader effort to expand newborn screening…
Activating a molecular recycling process called autophagy can improve motor function and extend lifespan in a nematode worm model of spinal muscular atrophy (SMA), a new study reports. “We provide the first evidence that pharmacological activation of autophagy enhanced neuromuscular performance in the severe C. elegans SMA model,” researchers…
For many people living with rare neuromuscular diseases, the most meaningful part of a recent Chicago gathering was not just the medical updates — it was finding a community that understood their daily struggles. The realization that they were not alone set the tone for MDA Engage: Chicago, a…
Sleep-related breathing problems were found to ease over time in children with spinal muscular atrophy (SMA) types 2 and 3 receiving the widely approved therapies Evrysdi (risdiplam) or Spinraza (nusinersen). That’s according to a new study from Sweden that followed children and teens with the two disease…
Muscle regeneration, the benefits of a specialized diet, physiotherapy, and assistive technologies are among top priorities for spinal muscular atrophy (SMA) research identified by patients, caregivers, and healthcare professionals from 22 countries in Europe. The top 10 priorities, developed through a series of surveys and a workshop, were published…
The brain has long been thought of as a singular organ, but a new study shows that the front and back parts of the brain actually develop completely independently of each other. This discovery may have key implications for research into diseases like spinal muscular atrophy (SMA), which affects…
A rapid, in-house genetic test allowed doctors in Japan to confirm a newborn’s diagnosis of spinal muscular atrophy (SMA) within hours and start treatment when the boy was 9 days old. The baby was readmitted to the hospital eight days after birth because of poor feeding and weight loss.
Children with spinal muscular atrophy (SMA) already living with symptoms experienced gains in motor abilities after receiving the gene therapy Zolgensma (onasemnogene abeparvovec-xioi), a small Brazilian study found. The real-world report tracked the outcomes of seven youngsters in the South American nation who were treated with Zolgensma only…
