News

After a “constructive and collaborative in-person” meeting with the U.S. Food and Drug Administration (FDA), Scholar Rock is now planning to once again seek approval of its muscle-strengthening agent apitegromab as a treatment for spinal muscular atrophy (SMA). That resubmission to the FDA is expected in…

The experimental therapy BIO101 may help improve muscle strength in people with spinal muscular atrophy (SMA), according to new research done in laboratory models. “We demonstrated that a daily oral administration of BIO101 can specifically improve muscular function in severe (infanthood) and mild (adulthood) … SMA mouse models,” the…

Treatment with Spinraza (nusinersen) may help stabilize lung function in people with spinal muscular atrophy (SMA) types 2 and 3, a study suggested. “Treatment of SMA types 2 and 3 with nusinersen may stabilize respiratory muscle strength and delay [noninvasive ventilation] dependence, despite progressive restrictive lung disease,” the…

First-line Zolgensma (onasemnogene abeparvovec-xioi) outperformed first-line Spinraza (nusinersen) in achieving a more sustained clinical response and reducing the need for supportive care among young children with spinal muscular atrophy (SMA) type 1, according to a head-to-head analysis. Still, both therapies led to comparable, clinically meaningful improvements in…

Mutations that cause spinal muscular atrophy (SMA) may trigger problems with how bone cells communicate, leading to abnormally weakened bones, according to a study done in mice and cells. The findings suggest that treatments aiming to normalize bone cell communication could help improve bone health in SMA patients, researchers…

Children with spinal muscular atrophy (SMA) type 1 can survive an episode of pediatric acute respiratory distress syndrome (pARDS), a type of life-threatening respiratory failure, according to a 12-year case series. While “pARDS remains a clinical challenge for SMA-1 children,” the researchers wrote, “the present series suggests survival is achievable…

People with spinal muscular atrophy (SMA) may show subtle abnormalities in eye movements, but these abnormalities are unlikely to be caused by weak muscles; instead, they likely arise due to changes in the brain, a study found. The study, “Central eye movement abnormalities in patients with SMA types…

Blocking a small RNA molecule called miR-140-3b can improve motor function in a mouse model of spinal muscular atrophy (SMA), a study suggested. Reducing miR-140-3b activity increased the levels of KIF5A, a protein involved in the transport of mitochondria (cellular powerhouses) inside nerve fibers. “We believe that KIF5A has…

Modifications that help certain genetic information travel to specific parts of nerve cells can ease motor symptoms and help normalize communication between nerves and muscles in a mouse model of spinal muscular atrophy (SMA), a study reported. SMA disrupts this nerve-muscle communication, which occurs at sites called neuromuscular junctions…

A one-time treatment designed to correct a single letter, or base, in the SMN2 gene increased levels of the survival motor neuron (SMN) protein — which is lacking in spinal muscular atrophy (SMA) — and prolonged survival in a mouse model of the disease when given before birth. The therapy…