title: Spinal muscular atrophy: a test case for drug development in orphan diseases.
authors.affiliation: lbain@nasw.org
authors.forename: Lisa J
authors.initials: LJ
authors.lastname: Bain
mesh_terms: 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