Issues in SMA clinical trial design. The International Coordinating Committee (ICC) for SMA Subcommittee on SMA Clinical Trial Design.
Kaufmann
P
P
Department of Neurology, Columbia University, New York, NY, USA, and Hammersmith Hospital, London, UK. pk88@columbia.edu
Muntoni
F
F
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