SMA in Mid-March: Clinical Practice Guidelines, Walking and Weakness in Children & More

SMA in Mid-March: Clinical Practice Guidelines, Walking and Weakness in Children & More

This month has seen a surge of publications focused on SMA treatments, with a trend towards optimizing various aspects of nusinersen treatment. Research has also continued in areas related to molecular mechanisms of the disease, clinical manifestations, and management...
SMA News for HCPs: Stasimon, nusinersen in adult patients, managing collapsing spine deformity & more

SMA News for HCPs: Stasimon, nusinersen in adult patients, managing collapsing spine deformity & more

Understanding SMA Stasimon contributes to the loss of sensory synapses and motor neuron death in a mouse model of spinal muscular atrophy.1 In this paper, the authors elucidate a mechanism of the motor neuron degeneration that occurs in SMA. Specifically, the authors...
FDA Approves AveXis’ Gene Therapy Zolgensma to Treat SMA in Children Up to Age 2

FDA Approves AveXis’ Gene Therapy Zolgensma to Treat SMA in Children Up to Age 2

Zolgensma, a first gene therapy for spinal muscular atrophy — and first for any chronic neurologic disease — is now an approved and potential “one-time” intravenous treatment for pre-symptomatic newborns through 2-year-olds with any type of SMA, the U.S. Food and Drug...