title: 209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7-9 November 2014, Heemskerk, The Netherlands.
authors.affiliation: Nemours Children's Hospital, Orlando, USA. Electronic address: rfinkel@nemours.org.
authors.forename: Richard
authors.initials: R
authors.lastname: Finkel
authors.affiliation: UCL Institute of Child Health, Dubowitz Neuromuscular Centre, London, UK.
authors.forename: Enrico
authors.initials: E
authors.lastname: Bertini
authors.affiliation: Bambino Gesu Children's Research Hospital, Rome, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Muntoni
authors.affiliation: Pediatric Neurology Unit, Catholic University, Rome, Italy.
authors.forename: Eugenio
authors.initials: E
authors.lastname: Mercuri
mesh_terms: D000818:Animals; D015415:Biomarkers / Q000378:metabolism; D002986:Clinical Trials as Topic; D004195:Disease Models, Animal; D006801:Humans; D009134:Muscular Atrophy, Spinal / Q000145:classification / Q000175:diagnosis* / Q000235:genetics / Q000628:therapy*; D009426:Netherlands; D017063:Outcome Assessment, Health Care
chemical_list: D015415:Biomarkers