Facioscapulohumeral muscular dystrophy is a rare genetic disorder that causes progressive muscle weakness and atrophy, predominantly in the face, shoulders, and upper arms, in an estimated 1 in 8,000 ...

Spinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor neuron disease with an onset during infancy that results in failure to achieve motor milestones and in death or the need for ...