Accelerating access to treatments for rare diseases

Nature Reviews Drug Discovery

Volumn 10, Issue 7, 2011, Pages 475-476

  Dunoyer, Marc ^a  

^a GLAXOSMITHKLINE   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

GLOBOTRIAOSYLCERAMIDE; ORPHAN DRUG;

CLINICAL TRIAL (TOPIC); DISEASE COURSE; DRUG DOSE REGIMEN; DRUG LEGISLATION; DRUG RESEARCH; DUCHENNE MUSCULAR DYSTROPHY; FRAMESHIFT MUTATION; GENE MUTATION; GENETIC HETEROGENEITY; HEALTH CARE ACCESS; HEALTH CARE DELIVERY; HEALTH CARE POLICY; HEALTH CARE QUALITY; HUMAN; ICHTHYOSIS; NOTE; PATHOPHYSIOLOGY; PHARMACOGENOMICS; PHENOTYPE; POLITICS; PRIORITY JOURNAL; RARE DISEASE; REIMBURSEMENT;

CLINICAL TRIALS AS TOPIC; DRUG APPROVAL; DRUG INDUSTRY; HUMANS; MOTIVATION; ORPHAN DRUG PRODUCTION; POLICY; RARE DISEASES; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 79960002064     PISSN: 14741776     EISSN: 14741784     Source Type: Journal    
DOI: 10.1038/nrd3493     Document Type: Note

References (2)

1. Braun, M. M. et al. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nature Rev. Drug Discov. 9, 519-522 (2010).
2. The Committee for Orphan Medicinal Products and the European Medicines Agency Scientific Secretariat. European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nature Rev. Drug Discov.10, 341-349 (2011).