Children with spinal muscular atrophy (SMA) have higher rates of emotional and behavioral challenges than children in the general population, even with today’s modern treatments, a new study reports. “In our treated SMA cohort, mental health challenges occurred across a variety of severities of motor function and created an…
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Before the end of this year, thanks to a new decision by the U.S. Food and Drug Administration (FDA), one-time gene therapy will be available to people of all ages with spinal muscular atrophy (SMA) in the United States. The first SMA gene therapy, Zolgensma (onasemnogene abeparvovec-xioi), was…
A high-dose regimen of nusinersen — the active ingredient in Spinraza — has been recommended for approval to treat spinal muscular atrophy (SMA) by the Committee for Medicinal Products for Human Use (CHMP), a branch of the European Medicines Agency. The decision was based on positive data from…
New treatments for spinal muscular atrophy (SMA) are helping many people live longer, fuller lives, changing what it means to grow up — and grow older — with the condition. SMA is now being managed across all stages of life, shifting care from palliative support toward long-term health and…
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…
