Spinal muscular atrophy

Spinal muscular atrophy (SMA), also called autosomal recessive proximal spinal muscular atrophy in order to distinguish it from other conditions with similar name – is a rare neuromuscular disorder characterised by loss of motor neurons and progressive muscle wasting, often leading to early death. Spinal muscular atrophy is an inherited disorder and is passed on in an autosomal recessive manner. As of 2016, no drugs for SMA have been approved, even as a number of therapeutics remain in late-stage clinical trials.

Spinal muscular atrophy

Spinal muscular atrophy (SMA), also called autosomal recessive proximal spinal muscular atrophy in order to distinguish it from other conditions with similar name – is a rare neuromuscular disorder characterised by loss of motor neurons and progressive muscle wasting, often leading to early death. Spinal muscular atrophy is an inherited disorder and is passed on in an autosomal recessive manner. As of 2016, no drugs for SMA have been approved, even as a number of therapeutics remain in late-stage clinical trials.