Molecule Similar to Evrysdi But Possibly Safe at Higher Doses Identified
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...
Muscle-directed Therapy SRK-015 Improves Motor Function in SMA Types 2, 3, Data Show
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...
SMA Type 3 Adults May Have Issues Regulating Component of Cell’s ‘Skeleton,’ Study Suggests
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...
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National...HCP — SMA News Today
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