Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

Nucleic Acid Therapeutics

Volumn 27, Issue 5, 2017, Pages 251-259

  Aartsma Rus, Annemieke ^a,b   Straub, Volker ^b   Hemmings, Robert ^c   Haas, Manuel ^d   Schlosser Weber, Gabriele ^e   Stoyanova Beninska, Violeta ^f   Mercuri, Eugenio ^g,h   Muntoni, Francesco ⁱ   Sepodes, Bruno ^j   Vroom, Elizabeth ^k   Balabanov, Pavel ^d  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^b NEWCASTLE UNIVERSITY   (United Kingdom)

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

^d EUROPEAN MEDICINES AGENCY   (United Kingdom)

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

^f MEDICINES EVALUATION BOARD   (Netherlands)

^g CATHOLIC UNIVERSITY   (Italy)

^h FONDAZIONE POLICLINICO UNIVERSITARIO A GEMELLI IRCCS   (Italy)

ⁱ UNIVERSITY COLLEGE LONDON   (United Kingdom)

^j RESEARCH INSTITUTE FOR MEDICINES IMED ULISBOA   (Portugal)

^k United Parent Projects Muscular Dystrophy   (Netherlands)

more..

Author keywords

exon skipping; oligonucleotides; regulatory approval

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN;

ANTISENSE THERAPY; DRUG APPROVAL; DUCHENNE MUSCULAR DYSTROPHY; EUROPEAN MEDICINES AGENCY; EXON; EXON SKIPPING; HUMAN; MUSCLE BIOPSY; PRIORITY JOURNAL; REVIEW; ANIMAL; DISEASE MODEL; FOOD AND DRUG ADMINISTRATION; GENE THERAPY; GENETICS; MOUSE; MUTATION; PROCEDURES; STAKEHOLDER ENGAGEMENT; UNITED STATES;

ANIMALS; DISEASE MODELS, ANIMAL; DRUG APPROVAL; DYSTROPHIN; EXONS; GENETIC THERAPY; HUMANS; MICE; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; OLIGONUCLEOTIDES, ANTISENSE; STAKEHOLDER PARTICIPATION; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 85030314866     PISSN: 21593337     EISSN: 21593345     Source Type: Journal    
DOI: 10.1089/nat.2017.0682     Document Type: Review

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