Scientists discover key mechanism for altered SMN2 gene processing
Scientists have identified a key mechanism responsible for altered processing in the body’s SMN2 gene that, in people with…
Articles by Steve Bryson, PhD
Current therapies aid motor function in SMA type 1 children in Spain
Current therapeutic approaches have helped to improve motor function in the majority of children with spinal muscular atrophy (SMA) type…
For SMA, growth factor NRG1-III improved nerve fibers in mice
Boosting the production of the growth factor protein NRG1-III improved motor nerve fiber development in the early stages in a…
SMA treatment responses predicted by certain microRNAs in early study
The MiR34 family of microRNAs represents a set of promising biomarkers to assess therapeutic response in spinal muscular atrophy…
Study supports mandatory newborn screening in Japan
A pilot study describes how researchers successfully implemented a newborn genetic screening in Osaka, Japan, to identify infants with…
Unusual SMN1 mutations detected with ultra-long DNA sequencing
Unusual mutations in the SMN1 gene, the underlying cause of spinal muscular atrophy (SMA), were detected in two young…
Gene editing to change SMN2 gene into SMN1 shows promise in mice
A single gene-editing treatment that converts the SMN2 gene into a functioning copy of SMN1 increased levels of the SMN…
Zolgensma into spinal canal helped older SMA type 2, 3 children
Older children with spinal muscular atrophy (SMA) type 2 or 3, ages 2–5, given a single injection of…
Evrysdi can affect sperm generation in males, animal studies find
High doses of Evrysdi (risdiplam), an approved daily therapy for spinal muscular atrophy (SMA), affected the development of…
Spinraza improves motor function in SMA children after loading doses
The motor function of children with spinal muscular atrophy (SMA) improves significantly in the early stages of treatment with…