The TREAT-NMD advisory committee for therapeutics (TACT): An innovative de-risking model to foster orphan drug development

Orphanet Journal of Rare Diseases

Volumn 10, Issue 1, 2015, Pages

  Heslop, Emma ^a   Csimma, Cristina ^b   Straub, Volker ^a   McCall, John ^c   Nagaraju, Kanneboyina ^d   Wagner, Kathryn R ^e   Caizergues, Didier ^f   Korinthenberg, Rudolf ^g   Flanigan, Kevin M ^h   Kaufmann, Petra ⁱ   McNeil, Elizabeth ⁱ   Mendell, Jerry ^h   Hesterlee, Sharon ^j   Wells, Dominic J ^k   Bushby, Kate ^a  

^a NEWCASTLE UNIVERSITY   (United Kingdom)

^b Cydan Developments Inc   (United States)

^c PharMac LLL   (United States)

^d CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^e JOHNS HOPKINS UNIVERSITY   (United States)

^f GÉNÉTHON   (France)

^g UNIVERSITY OF FREIBURG   (Germany)

^h NATIONWIDE CHILDREN S HOSPITAL   (United States)

ⁱ NATIONAL INSTITUTES OF HEALTH   (United States)

^j Parent Project Muscular Dystrophy   (United States)

^k ROYAL VETERINARY COLLEGE   (United Kingdom)

more..

Author keywords

De risking; Drug development; Neuromuscular disease; Rare disease; Review

Indexed keywords

ORPHAN DRUG;

ADVISORY COMMITTEE; ARTICLE; EUROPEAN MEDICINES AGENCY; FOOD AND DRUG ADMINISTRATION; FUNDING; HEALTH CARE UTILIZATION; HUMAN; MEDICAL PROCEDURES; NEUROMUSCULAR DISEASE; PHARMACY AND THERAPEUTICS COMMITTEE; ANIMAL; DRUG MANUFACTURE; NEUROMUSCULAR DISEASES; RARE DISEASES; UNITED STATES;

ADVISORY COMMITTEES; ANIMALS; HUMANS; NEUROMUSCULAR DISEASES; ORPHAN DRUG PRODUCTION; RARE DISEASES; UNITED STATES;

EID: 84928633900     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-015-0258-1     Document Type: Article

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