title: European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene.
authors.affiliation: European Medicines Agency, London, United Kingdom. Electronic address: manuel.haas@ema.europa.eu.
authors.forename: Manuel
authors.initials: M
authors.lastname: Haas
authors.affiliation: European Medicines Agency, London, United Kingdom.
authors.forename: Viktor
authors.initials: V
authors.lastname: Vlcek
authors.affiliation: European Medicines Agency, London, United Kingdom.
authors.forename: Pavel
authors.initials: P
authors.lastname: Balabanov
authors.affiliation: Läkemedelsverket, Medical Products Agency, Uppsala, Sweden.
authors.forename: Tomas
authors.initials: T
authors.lastname: Salmonson
authors.affiliation: Hôpital Maison Blanche, CHU de Reims, Reims, France.
authors.forename: Serge
authors.initials: S
authors.lastname: Bakchine
authors.affiliation: Medicines and Healthcare Products Regulatory Agency, London, United Kingdom.
authors.forename: Greg
authors.initials: G
authors.lastname: Markey
authors.affiliation: Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn, Germany.
authors.forename: Martina
authors.initials: M
authors.lastname: Weise
authors.affiliation: Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn, Germany.
authors.forename: Gabriele
authors.initials: G
authors.lastname: Schlosser-Weber
authors.affiliation: Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn, Germany.
authors.forename: Henning
authors.initials: H
authors.lastname: Brohmann
authors.affiliation: Agencia Española de Medicamentos y Productos Sanitarios, Madrid, Spain.
authors.forename: Concepcion Prieto
authors.initials: CP
authors.lastname: Yerro
authors.affiliation: Agencia Española de Medicamentos y Productos Sanitarios, Madrid, Spain.
authors.forename: Macarena Rodriguez
authors.initials: MR
authors.lastname: Mendizabal
authors.affiliation: Medicines Evaluation Board, Utrecht, The Netherlands.
authors.forename: Violeta
authors.initials: V
authors.lastname: Stoyanova-Beninska
authors.affiliation: Medicines Evaluation Board, Utrecht, The Netherlands.
authors.forename: Hans L
authors.initials: HL
authors.lastname: Hillege
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D017322:Clinical Trials, Phase II as Topic; D018389:Codon, Nonsense; D017277:Drug Approval; D016189:Dystrophin / Q000235:genetics*; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000235:genetics*; D010069:Oxadiazoles / Q000627:therapeutic use*; D016032:Randomized Controlled Trials as Topic; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D018389:Codon, Nonsense; D016189:Dystrophin; D010069:Oxadiazoles; C515878:ataluren