Scholar Rock has successfully removed a manufacturing facility from its application to the U.S. Food and Drug Administration (FDA) seeking approval of apitegromab, an experimental muscle-strengthening treatment for spinal muscular atrophy (SMA). The…
Treatment before birth with RG7916, a compound similar to Evrysdi (risdiplam), improved weight, muscle health, motor function, and survival in a mouse model of severe spinal muscular atrophy (SMA), a study found. However, treatment given…
A woman with spinal muscular atrophy (SMA) type 3 experienced a mild decline in motor function after pausing Spinraza (nusinersen) during her pregnancy. After she restarted treatment following delivery, however, her motor function stabilized and her walking endurance…
A panel of experts has developed the first U.K. national consensus recommendations for preventing and managing hip problems in children with spinal muscular atrophy (SMA), aiming to support earlier monitoring and more consistent care…
Children with spinal muscular atrophy (SMA) frequently develop what are known as musculoskeletal deformities, or bone and muscle-related problems that include scoliosis, a sideways curvature of the spine, and hip dislocations, according to a new study…
Throughout 2025, the team at SMA News Today has brought our audience the latest advances in spinal muscular atrophy (SMA), from emerging treatments and clinical trial updates to real-world research. As the year comes to a close, we’ve…
A new review of studies from six countries reveals that incorporating spinal muscular atrophy (SMA) into newborn screening (NBS) programs yields clinical improvements and reduces healthcare spending, particularly when paired with presymptomatic treatment. “These results collectively…
SMA Europe has launched an online tool designed to help patients, doctors, and others in the spinal muscular atrophy (SMA) community more easily explore ongoing clinical trials across Europe and worldwide. The organization said it aims…
The “Tales from the Magic Keep” exergame — a video game that uses body movements to promote physical activity — proved to be a feasible and engaging home-based tool for children and adolescents with spinal muscular…
Spinraza (nusinersen) helps correct a key amino acid deficiency in people with a severe form of spinal muscular atrophy (SMA), a study has found. Researchers found that the treatment restores levels of L-arginine, an amino…
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