title: Issues in SMA clinical trial design. The International Coordinating Committee (ICC) for SMA Subcommittee on SMA Clinical Trial Design.
authors.affiliation: Department of Neurology, Columbia University, New York, NY, USA, and Hammersmith Hospital, London, UK. pk88@columbia.edu
authors.forename: P
authors.initials: P
authors.lastname: Kaufmann
authors.forename: F
authors.initials: F
authors.lastname: Muntoni
mesh_terms: D002986:Clinical Trials as Topic; D004219:District of Columbia; D006801:Humans; D007391:International Cooperation; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy* / Q000503:physiopathology / Q000523:psychology; D017063:Outcome Assessment, Health Care; D011788:Quality of Life