Researchers compared children with types 1 and 2 spinal muscular atrophy (SMA) in terms of body composition and resting energy expenditure, and found those measures — for now — are the best to use when attempting to determine their appropriate dietary needs. Body mass and body weight index Z-scores (a measure…
A Phase 2 clinical trial investigating a potential treatment, CK-2127107, in people with spinal muscular atrophy (SMA), ages 12 years and older, is currently recruiting patients in the U.S. with varying degrees of disease severity (Types 2, 3, or 4). CK-2127107 is an investigational drug candidate designed for skeletal muscle and other debilitating neuromuscular…
Researchers from The Jackson Laboratory in Maine, in collaboration with Isis Pharmaceuticals, tested a novel therapeutic candidate (antisense oligonucleotides) in genetically engineered mice with spinal muscular atrophy (SMA), observing it could restore some functionality of motor neurons. The study entitled “Systemic, postsymptomatic antisense oligonucleotide rescues motor…