Sma clinical data, outcome measures and registries

Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by paralysis and muscle weakness and can affect both children and adults. The first FDA approved SMA therapeutic, Spinraza, improves SMA patients' symptoms and motor function. It is expected that increased lifespan will lead to a shift from the severe infantile SMA population to a milder SMA adult population. It is unknown whether such lifespan extension will reveal new, previously unknown defects that could arise with age, as mild mouse models of SMA are currently lacking.

Source: Neuromuscular Disorders - September 6, 2018 Category: Neurology Authors: M. Deguise, A. Beauvais, A. Tiernay, B. Paul, E. McFall, Y. De Repentigny, R. Kothary Source Type: research