Treatment with Spinraza (nusinersen) was linked to measurable changes in a range of cerebrospinal fluid (CSF) biomarkers in people with spinal muscular atrophy (SMA), some of which were associated with improvements in motor function, according to…
Evidence-based guidelines have been developed for newborn screening (NBS), diagnosis, and early management of spinal muscular atrophy (SMA) in Australia and New Zealand to help ensure that all affected children are identified and treated as early as…
Newborn screening (NBS) for spinal muscular atrophy (SMA) is widely supported among families whose babies were screened, SMA families and adults living with the disease, healthcare professionals, and the public, according to a large U.K. survey. The potential…
A review study found that the neuromuscular junction (NMJ), the site where nerves connect to the muscles they control, fails to mature normally soon after birth in people with spinal muscular atrophy (SMA), leaving it structurally underdeveloped…
Whole-body quantitative MRI, or qMRI, can track long-term subtle muscle changes throughout the body among adults with spinal muscular atrophy (SMA), despite treatment and clinical stability, a new study reported. In fact, qMRI was seen…
Scholar Rock plans to resubmit its application to the U.S. Food and Drug Administration (FDA) seeking approval for apitegromab, an investigational muscle-strengthening therapy for spinal muscular atrophy (SMA). The submission is contingent on…
Damage to blood vessels in the spinal cord may play a role in nerve cell loss in spinal muscular atrophy (SMA), a new study suggests. A detailed examination of spinal cord tissue from infants…
Children with spinal muscular atrophy (SMA) who are nonambulatory, or unable to walk, have weaker upper leg bones — as assessed by tests of bone mineral density, or BMD — compared with nonambulatory…
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…
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…
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