Clinical research for rare disease: Opportunities, challenges, and solutions

Molecular Genetics and Metabolism

Volumn 96, Issue 1, 2009, Pages 20-26

  Griggs, Robert C ^a   Batshaw, Mark ^b   Dunkle, Mary ^c   Gopal Srivastava, Rashmi ^d   Kaye, Edward ^e   Krischer, Jeffrey ^f   Nguyen, Tan ^g   Paulus, Kathleen ^f   Merkel, Peter A ^h  

^a UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

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

^c National Organization for Rare Disorders   (United States)

^d NATIONAL INSTITUTES OF HEALTH   (United States)

^e GENZYME CORPORATION   (United States)

^f UNIVERSITY OF SOUTH FLORIDA   (United States)

^g CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^h Vasculitis Center   (United States)

Author keywords

Clinical trials; Patient advocacy groups (PAGs); Rare diseases; Research training

Indexed keywords

ANTINEOPLASTIC AGENT; ORPHAN DRUG;

ARTICLE; CLINICAL RESEARCH; DEVICE; DRUG APPROVAL; DRUG INDUSTRY; DRUG LEGISLATION; DRUG MARKETING; FOOD AND DRUG ADMINISTRATION; INTERNET; MEDICAL INFORMATION; METABOLIC DISORDER; MOLECULAR GENETICS; PATHOPHYSIOLOGY; PATIENT ADVOCACY; PATIENT PARTICIPATION; PATIENT SELECTION; PRESCRIPTION; PRIORITY JOURNAL; RARE DISEASE; SCIENTIFIC LITERATURE;

BIOMEDICAL RESEARCH; CLINICAL TRIALS AS TOPIC; DRUG DESIGN; FINANCING, GOVERNMENT; HUMANS; ORPHAN DRUG PRODUCTION; PUBLIC-PRIVATE SECTOR PARTNERSHIPS; RARE DISEASES; UNITED STATES;

EID: 57649109461     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2008.10.003     Document Type: Article

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