title: Recommendations for myasthenia gravis clinical trials.
abstract: The recommendations for clinical research standards published in 2000 by a task force of the Medical Scientific Advisory Board (MSAB) of the Myasthenia Gravis Foundation of America (MGFA) were largely successful in introducing greater uniformity in the recording and reporting of MG clinical trials. Recognizing that changes in clinical trial design and implementation may increase the likelihood that new therapies are developed for MG, the MGFA MSAB Task Force here presents updated recommendations for the design and implementation of clinical trials in MG, including (a) the use of a quantitative measure, such as the MG-Composite, that is weighted for clinical significance and incorporates patient reported outcomes; (b) consideration of nontrial strategies; and (c) development of biomarkers that support mechanistic studies of pharmacotherapies. The hope is that these updated task force recommendations will expedite the development and acceptance of more effective and less noxious therapies for MG.
authors.affiliation: Department of Neurology, University of Miami School of Medicine, Miami, Florida, USA.
authors.forename: Michael
authors.initials: M
authors.lastname: Benatar
authors.forename: Donald B
authors.initials: DB
authors.lastname: Sanders
authors.forename: Ted M
authors.initials: TM
authors.lastname: Burns
authors.forename: Gary R
authors.initials: GR
authors.lastname: Cutter
authors.forename: Jeffrey T
authors.initials: JT
authors.lastname: Guptill
authors.forename: Fulvio
authors.initials: F
authors.lastname: Baggi
authors.forename: Henry J
authors.initials: HJ
authors.lastname: Kaminski
authors.forename: Renato
authors.initials: R
authors.lastname: Mantegazza
authors.forename: Matthew N
authors.initials: MN
authors.lastname: Meriggioli
authors.forename: Joanne
authors.initials: J
authors.lastname: Quan
authors.forename: Gil I
authors.initials: GI
authors.lastname: Wolfe
mesh_terms: D026683:Advisory Committees; D015415:Biomarkers; D002986:Clinical Trials as Topic / Q000592:standards*; D006801:Humans; D009157:Myasthenia Gravis / Q000628:therapy*; D017063:Outcome Assessment, Health Care; D012107:Research Design / Q000592:standards*; D012955:Societies, Medical; D014481:United States
chemical_list: D015415:Biomarkers