Orphan drug development: the increasing role of clinical pharmacology

Journal of Pharmacokinetics and Pharmacodynamics

Volumn 46, Issue 5, 2019, Pages 395-409

  Ahmed, Mariam A ^a,d   Okour, Malek ^b   Brundage, Richard ^a,c   Kartha, Reena V ^a,c  

^a UNIVERSITY OF MINNESOTA   (United States)

^b GLAXOSMITHKLINE   (United States)

^c UNIVERSITY OF MINNESOTA   (United States)

^d NONE   (United States)

Author keywords

Model informed drug discovery and development; Modeling and simulations; Orphan drugs; Rare diseases

Indexed keywords

BOSENTAN; DOMAGROZUMAB; DORNASE ALFA; ECULIZUMAB; EMICIZUMAB; INFLIXIMAB; IVACAFTOR; LORENZO OIL; ORPHAN DRUG; SILDENAFIL; TETRABENAZINE;

CLINICAL PHARMACOLOGY; CLINICAL TRIAL (TOPIC); CYSTIC FIBROSIS; DOSE CALCULATION; DRUG APPROVAL; DRUG INDICATION; HUMAN; NONHUMAN; PRIORITY JOURNAL; PULMONARY HYPERTENSION; REVIEW; SIMULATION; STUDY DESIGN; DRUG MANUFACTURE; FOOD AND DRUG ADMINISTRATION; PROCEDURES; UNITED STATES;

DRUG APPROVAL; HUMANS; ORPHAN DRUG PRODUCTION; PHARMACOLOGY, CLINICAL; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 85069529331     PISSN: 1567567X     EISSN: 15738744     Source Type: Journal    
DOI: 10.1007/s10928-019-09646-3     Document Type: Review

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