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

Neuromuscular Disorders

Volumn 25, Issue 1, 2015, Pages 5-13

  Haas, Manuel ^a   Vlcek, Viktor ^a   Balabanov, Pavel ^a   Salmonson, Tomas ^b   Bakchine, Serge ^c   Markey, Greg ^d   Weise, Martina ^e   Schlosser Weber, Gabriele ^e   Brohmann, Henning ^e   Yerro, Concepcion Prieto ^f   Mendizabal, Macarena Rodriguez ^f   Stoyanova Beninska, Violeta ^g   Hillege, Hans L ^g  

^a EUROPEAN MEDICINES AGENCY   (United Kingdom)

^b MEDICAL PRODUCTS AGENCY   (Sweden)

^c HÔPITAL MAISON BLANCHE   (France)

^d MEDICINES AND HEALTHCARE PRODUCTS REGULATORY AGENCY   (United Kingdom)

^e Federal Institute for Drugs and Medical Devices Bundesinstitut für Arzneimittel und Medizinprodukte   (Germany)

^f Agencia Espanola de Medicamentos y Productos Sanitarios   (Spain)

^g MEDICINES EVALUATION BOARD   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

ATALUREN; DYSTROPHIN; PLACEBO; OXADIAZOLE DERIVATIVE; STOP CODON;

ABDOMINAL PAIN; APPETITE DISORDER; ARTHRALGIA; ARTICLE; ASTHENIA; BACKACHE; CHILD; COUGHING; DIARRHEA; DIZZINESS; DRUG APPROVAL; DRUG EFFICACY; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; DYSTROPHIN GENE; EAR INFECTION; EUROPE; FEVER; FLATULENCE; GASTROENTERITIS; HEADACHE; HUMAN; INFLUENZA; MUSCLE SPASM; MUSCLE WEAKNESS; MYOMETRY; NAUSEA; NONHUMAN; NONSENSE MUTATION; NOSE OBSTRUCTION; OROPHARYNX PAIN; PAIN; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PRESCHOOL CHILD; RANDOMIZED CONTROLLED TRIAL (TOPIC); RASH; RHINITIS; RHINOPHARYNGITIS; RHINORRHEA; RISK BENEFIT ANALYSIS; SCAR; SIDE EFFECT; STOMACH DISCOMFORT; UPPER RESPIRATORY TRACT INFECTION; VIRAL GASTROENTERITIS; VOMITING; ADOLESCENT; ADULT; GENETICS; MALE; MUSCULAR DYSTROPHY, DUCHENNE; STOP CODON; TREATMENT OUTCOME; YOUNG ADULT;

ADOLESCENT; ADULT; CHILD; CHILD, PRESCHOOL; CLINICAL TRIALS, PHASE II AS TOPIC; CODON, NONSENSE; DRUG APPROVAL; DYSTROPHIN; HUMANS; MALE; MUSCULAR DYSTROPHY, DUCHENNE; OXADIAZOLES; RANDOMIZED CONTROLLED TRIALS AS TOPIC; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84919625145     PISSN: 09608966     EISSN: 18732364     Source Type: Journal    
DOI: 10.1016/j.nmd.2014.11.011     Document Type: Article

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