Manipulating Protein and Gene Seen to Improve SMA Motor Symptoms in Animal Study
Lowering levels of a newly identified protein, in combination with a splice-modifying therapy, improved symptoms and survival in animal models of spinal muscular atrophy (SMA), according to a recent study. The finding suggests that targeting the factor — called neuronal calcium sensor neurocalcin delta (NCALD) — could be beneficial for SMA patients,…