Rare Genetic

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Spinal Muscular Atrophy Emerging therapies

Spinal Muscular Atrophy (SMA) is a rare genetic and neuromuscular condition which affects mainly children. It has several distinct types such as type 1 (Werdnig-Hoffmann disease), type 2 (Dubowitz disease), type 3 (Kugelberg-Welander disease) and type 4 (Adult onset). SMN1 (Survival Motor Neuron 1) is the primary ge...

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