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Spinal Muscular Atrophy

Welcome to the SMA learning Zone

Welcome to the Spinal Muscular Atrophy Learning Zone

Spinal muscular atrophy (SMA) is a heterogeneous, genetic, neuromuscular disease with complications that impact multiple organs. Patients with SMA have insufficient levels of the survival of motor neuron (SMN) protein which results in motor neuron loss. The loss of motor neurons in patients with SMA results in significant loss of motor function. Click below to watch Dr Giovanni Baranello, one of the leading experts in the treatment of SMA, explain improvement of motor function in patients with SMA.

This Learning Zone provides an overview of SMA including
pathophysiologygenetics of the underlying disease, disease burden, diagnosis, treatment guidelines and disease management. Also covered are the most recent regulatory approvals, new developments in research and ongoing clinical trials that impact disease management and treatment.