Most studies of children with SMA1 have focused on physical function, not their intellectual and language abilities. Children with SMA1 have general intelligence and language comprehension in the ...

Spinal muscular atrophy affects the development of the nervous system more profoundly than was previously thought, a new ...

Speech impairment was primarily linked to motor impairment, according to a study examining the development trajectory of children with spinal muscular atrophy. A recent study from Italy examined ...

A case report shows a baby with SMA met typical motor milestones at 10 months after a planned early C-section helped ...

SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves ...

SOUTH PLAINFIELD, N.J., March 16, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced two-year data from Part 2 of the SUNFISH trial evaluating Evrysdi™ (risdiplam) in children ...

SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves ...

The MScanFit method assessed changes in motor unit configuration in adult patients undergoing treatment for spinal muscular atrophy (SMA). In recent years, spinal muscular atrophy (SMA) diagnosis and ...

First and only muscle-targeted SMA therapy to demonstrate statistically significant, clinically meaningful improvements on the gold-standard HFMSE scale (p=0.019) versus placebo, with consistent ...

Basel, March 24, 2020 – AveXis, a Novartis company, today announced a one-time infusion of Zolgensma® (onasemnogene abeparvovec-xioi) showed rapid, significant and clinically meaningful therapeutic ...