Drug development for exceptionally rare metabolic diseases: Challenging but not impossible

Orphanet Journal of Rare Diseases

Volumn 8, Issue 1, 2013, Pages

  Putzeist, Michelle ^a,b   Mantel Teeuwisse, Aukje K ^a   Wied, Christine C Gispen De ^b   Hoes, Arno W ^c   Leufkens, Hubert G M ^a,b   De Vrueh, Remco L A ^d  

^a UTRECHT UNIVERSITY   (Netherlands)

^b MEDICINES EVALUATION BOARD   (Netherlands)

^c UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^d Rare disease matters   (Netherlands)

Author keywords

Inherited metabolic disease; Orphan medicinal product; Preclinical proof of concept; Prevalence; Rare disease

Indexed keywords

ORPHAN DRUG;

ANIMAL MODEL; ARTICLE; CARBOHYDRATE METABOLISM; ENERGY METABOLISM; FOOD AND DRUG ADMINISTRATION; GENE FUNCTION; INTOXICATION; METABOLIC DISORDER; PREVALENCE; PROGNOSIS; RISK FACTOR;

DRUG APPROVAL; EUROPE; FEMALE; HUMANS; MALE; METABOLIC DISEASES; ORPHAN DRUG PRODUCTION; RARE DISEASES; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84887514882     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-8-179     Document Type: Article

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