Pressure for drug development in lysosomal storage disorders - A quantitative analysis thirty years beyond the US orphan drug act

Orphanet Journal of Rare Diseases

Volumn 10, Issue 1, 2015, Pages

  Mechler, Konstantin ^a   Mountford, William K ^b   Hoffmann, Georg F ^c   Ries, Markus ^c  

^a UNIVERSITY OF HEIDELBERG   (Germany)

^b UNIVERSITY OF NORTH CAROLINA WILMINGTON   (United States)

^c UNIVERSITY HOSPITAL HEIDELBERG   (Germany)

Author keywords

Drug development; Orphan disease; Small clinical trials

Indexed keywords

BIOLOGICAL MARKER; ORPHAN DRUG;

ARTICLE; CLINICAL TRIAL (TOPIC); DATA BASE; DRUG APPROVAL; DRUG LEGISLATION; ENZYME REPLACEMENT; FOOD AND DRUG ADMINISTRATION; GENE THERAPY; HUMAN; LYSOSOME STORAGE DISEASE; MEDICAL TECHNOLOGY; NEUROLOGIC DISEASE; PREVALENCE; QUANTITATIVE ANALYSIS; STUDY DESIGN; DRUG DEVELOPMENT; DRUG INDUSTRY; DRUG MANUFACTURE; ECONOMICS; LEGISLATION AND JURISPRUDENCE; LYSOSOMAL STORAGE DISEASES; RARE DISEASES; STATISTICS AND NUMERICAL DATA; UNITED STATES;

DRUG APPROVAL; DRUG DISCOVERY; DRUG INDUSTRY; HUMANS; LYSOSOMAL STORAGE DISEASES; ORPHAN DRUG PRODUCTION; RARE DISEASES; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84928018148     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-015-0262-5     Document Type: Article

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