Stakeholder cooperation to overcome challenges in orphan medicine development: The example of Duchenne muscular dystrophy

The Lancet Neurology

Volumn 15, Issue 8, 2016, Pages 882-890

  Straub, Volker ^a   Balabanov, Pavel ^b   Bushby, Kate ^a   Ensini, Monica ^a   Goemans, Nathalie ^c   De Luca, Annamaria ^d   Pereda, Alejandra ^e   Hemmings, Robert ^f   Campion, Giles ^g   Kaye, Edward ^h   Arechavala Gomeza, Virginia ⁱ   Goyenvalle, Aurelie ^j   Niks, Erik ^k   Veldhuizen, Olav ^a   Furlong, Pat ^l   Stoyanova Beninska, Violeta ^m   Wood, Matthew J ⁿ   Johnson, Alex ^o   Mercuri, Eugenio ^p   Muntoni, Francesco ^q   Sepodes, Bruno ^r   Haas, Manuel ^b   Vroom, Elizabeth ^s   Aartsma Rus, Annemieke ^a   more..

^a NEWCASTLE UNIVERSITY   (United Kingdom)

^b EUROPEAN MEDICINES AGENCY   (United Kingdom)

^c UNIVERSITY HOSPITALS LEUVEN   (Belgium)

^d UNIVERSITY OF BARI   (Italy)

^e Duchenne Parent Project   (Spain)

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

^g BIOMARIN PHARMACEUTICAL INC   (United States)

^h AVI BIOPHARMA INC   (United States)

ⁱ BIOCRUCES BIZKAIA HEALTH RESEARCH INSTITUTE   (Spain)

^j LABORATOIRE DES SCIENCES DU CLIMAT ET DE L ENVIRONNEMENT   (France)

^k LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^l Parent Project Muscular Dystrophy   (United States)

^m MEDICINES EVALUATION BOARD   (Netherlands)

ⁿ UNIVERSITY OF OXFORD   (United Kingdom)

^o Joining Jack   (United Kingdom)

^p CATHOLIC UNIVERSITY   (Italy)

^q MEDICAL RESEARCH COUNCIL   (United Kingdom)

^r RESEARCH INSTITUTE FOR MEDICINES IMED ULISBOA   (Portugal)

^s United Parent Projects Muscular Dystrophy   (Netherlands)

more..

Author keywords

[No Author keywords available]

Indexed keywords

BIOLOGICAL MARKER; DYSTROPHIN;

DUCHENNE MUSCULAR DYSTROPHY; HUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; OUTCOME ASSESSMENT; PRIORITY JOURNAL; REVIEW; RISK BENEFIT ANALYSIS; TREATMENT PLANNING; DRUG APPROVAL; DRUG MANUFACTURE; GENE THERAPY; GENETICS; MUSCULAR DYSTROPHY, DUCHENNE; PUBLIC-PRIVATE PARTNERSHIP;

DRUG APPROVAL; GENETIC THERAPY; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; ORPHAN DRUG PRODUCTION; OUTCOME ASSESSMENT (HEALTH CARE); PUBLIC-PRIVATE SECTOR PARTNERSHIPS;

EID: 84973316149     PISSN: 14744422     EISSN: 14744465     Source Type: Journal    
DOI: 10.1016/S1474-4422(16)30035-7     Document Type: Review

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