Spinal Muscular Atrophy (SMA)

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Is Scoliosis Surgery in Patients With SMA Evidence-Based?

Spinal muscular atrophy (SMA) is a disorder characterized by muscle atrophy and weakness. It is inherited in an autosomal recessive manner and is caused by mutations/deletions in the SMN1 gene, which ensures adequate levels of survival motor neuron proteins in the body.  Patients with SMA tend to experience proximal muscle weakness, especially in the lower…

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Patient Perspectives

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SMA Features

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Rare Care Podcast

An interview with SMA researcher Adrian Krainer, PhD, from the Cold Spring Harbor Laboratory.