Liver-specific SMN1 gene therapy both improved liver functioning, and also reduced other SMA-like symptoms in a mouse model ...

New research suggests previous assumptions about the pathogenesis of spinal muscular atrophy (SMA) are inadequate.

Muscle fibers in children with SMA type 1 show substantial differences in components of the myosin protein called heavy ...

Scholar Rock Holding Corp (SRRK) reports significant progress in its SAPPHIRE study and financial growth, setting the stage ...

However, by the time this protein aggregation develops, the damage is well underway ... Interestingly, SmD1 is a survival of ...

SMA is a rare neuromuscular disease caused by a mutation in the SMN1 gene, which codes for a protein called SMN that is essential for the survival of motor neurons. The mutation means that no ...

Approximately 5,000 people in the U.S. develop amyotrophic lateral sclerosis (ALS) each year. On average, they survive for only two to five years after being diagnosed, according to the Centers for ...

This important manuscript provides insights into the competition between Splicing Factor 1 (SF1) and Quaking (QKI) for binding at the ACUAA branch point sequence in a model intron, regulating exon ...

A retrospective, single-center cohort study utilized liver ultrasound, serum collection, and proteome analysis to draw associations between spinal motor neuron (SMN) protein depletion and ...

What's more, the SmD1 protein is part of SMN, a multiprotein complex. SMN dysfunction is implicated in another neurodegenerative disorder, spinal muscular atrophy. “We're intrigued because there are ...

If you want to lose weight in a healthy, sustainable way, it's essential to get adequate levels of protein in your daily diet. For one, consuming healthy protein helps you feel satiated for longer ...