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...
SMA in Early January: Coaxial Curved-Needle Technique, Diagnostic Accuracy of MRI & More
2019 was a year of significant progress in our understanding of SMA and how to best address it – clinically and otherwise. Below is a roundup of some of the final SMA data shared in late 2019. Understanding SMA The classical complement pathway mediates...
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...
Reldesemtiv Improves Muscle Strength in SMA Patients, Trial Data Show
Reldesemtiv improved muscle strength for walking and respiration in a dose-dependent manner in spinal muscular atrophy (SMA) patients, data from a Phase 2 clinical trial show. As a fast muscle activator, reldesemtiv will not treat the underlying causes of SMA — a lack...HCP — SMA News Today
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