Olesoxime, a small molecule once considered a potential treatment, failed to slow motor and respiratory decline in people with spinal muscular atrophy (SMA) types 2 and 3 who were unable to walk, according to final results from an extension Phase 2 clinical trial. Data covering nearly 2.5 years of treatment…
By taking advantage of a natural process of gene silencing, a new gene therapy approach appears to prevent the toxicity to dorsal root ganglion (DRG) — a specific cluster of sensory neurons — seen in non-human primates during gene therapy studies for neurological disorders, researchers report. The approach successfully protected…
Spinraza (nusinersen) effectively preserves respiratory muscle strength in addition to motor function in children with spinal muscular atrophy (SMA) type 2, according to a real-life, small study in France. Larger studies are needed to confirm these findings, and to determine the usefulness of age-adapted respiratory muscle assessment as an efficacy measure…
A newly identified small molecule able to cross into the brain — named TEC-1 — significantly eased disease severity and prolonged survival in a mouse model of spinal muscular atrophy (SMA), a study from Japan reported. TEC-1 was reported to work in ways similar to Genentech’s oral SMA…
The Australian Medical Services Advisory Committee (MSAC) has recommended that the costs of screening for mutations known to cause spinal muscular atrophy (SMA), cystic fibrosis (CF) and fragile X syndrome be open to reimbursement for couples planning or in the early stages of pregnancy. Its favorable opinion…
England’s National Institute for Health and Care Excellence (NICE) has begun a review process to decide whether to include spinal muscular atrophy (SMA) type 3 patients who are unable to walk unaided in the country’s conditional reimbursement program for Spinraza (nusinersen). This conditional reimbursement program is part of a managed…
SRK-015, Scholar Rock’s muscle-targeted therapy, safely and effectively improved motor function in children and young adults with spinal muscular atrophy (SMA) types 2 and 3, six-month interim data from a Phase 2 trial show. These benefits were more pronounced with higher doses of SRK-015 and, not surprisingly, in those…
Adults with spinal muscular atrophy (SMA) type 3 have significantly lower levels of several molecules that help regulate actin, one of the major components of the cell’s cytoskeleton, according to a study. These findings, from blood and connective tissue cells, add to information from a previous study showing…
About one year of treatment with Spinraza (nusinersen) gradually and significantly improves motor function in adults with spinal muscular atrophy (SMA) type 3 — especially those unable to walk without assistance, according to a large and real-life study in Italy. Adults with type 2 disease, in turn,…
Evrysdi (risdiplam), Roche’s oral therapy for spinal muscular atrophy (SMA), will be available to select patients in the U.K. before its potential regulatory approval through an Early Access to Medicines Scheme (EAMS). Eligible patients include those with type 1 and 2 disease, ages 2…
