SMA Mutations Impact Development, Protein Production in Prenatal Mice
An analysis of prenatal mice embryos carrying genetic mutations that cause severe cases of spinal…
Articles by Steve Bryson, PhD
Gene Therapy Reduces Disease Severity in SMA Mouse Model, Study Shows
Gene therapy that increased the production of a protein called DOK7 in a spinal muscular atrophy (SMA)…
4-aminopyridine Fails to Improve Muscle Function in SMA Type 3 Patients, Small Trial Finds
Treatment with the molecule 4-aminopyridine (4-AP) failed to improve the motor function, endurance, and muscle strength…
GridPad Trilogy Communication Device Aids People With Speech and Motor Disorders
Control Bionics has announced the launch of the GridPad Trilogy, an…
Blocking Calpain Enzyme Found to Increase SMN Protein in Preclinical Models
In cell- and mouse-based models of spinal muscular atrophy (SMA), reduced levels of…
Spinraza Eases Fatigue in Adults With SMA But Benefit Wanes, Study Finds
Spinraza significantly eased fatigue in adults with spinal muscular atrophy (SMA)…
SMA Treatment and Screening Programs ‘Essential’ in Pandemic, Panel Says
Continuing treatment for spinal muscular atrophy (SMA) is essential and should not be considered…
Range of SMA Severity Not Explained by Mutations in SMN-Related Genes, Study Says
Mutations in genes that interact with the SMN protein, which is lacking or present at low levels in spinal…
Evidence Points to Benefits of Newborn Screening for SMA, German Researchers Say
Researchers in Germany have found that newborn screening for spinal muscular atrophy (SMA) would likely be beneficial, based on…