Spinal muscular atrophy: a test case for drug development in orphan diseases.
Bain
Lisa J
LJ
lbain@nasw.org
D015195:Drug Design; D004353:Drug Evaluation, Preclinical; D006801:Humans; D009046:Motor Neurons / Q000187:drug effects; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy*; D009965:Orphan Drug Production; D010602:Pharmacopoeias as Topic