Periodic Reporting for period 1 - SMABEYOND (Spinal Muscular Atrophy (SMA) beyond motoneuron degeneration: multi-system aspects)

Spinal Muscular Atrophy (SMA) is a monogenic motoneuron disease with a neuromuscular phenotype resulting in infant death in severe cases. Besides motoneurons in the central nervous system (CNS), there is growing evidence of an involvement of peripheral organs. SMA is caused by...