Therapies for inborn errors of metabolism: what has the orphan drug act delivered?

Pediatrics

Volumn 126, Issue 1, 2010, Pages 101-106

  Talele, Sonali S ^a,b   Xu, Kui ^a   Pariser, Anne R ^a   Braun, M Miles ^a   Farag El Massah, Sheiren ^a,c   Phillips, M Ian ^b   Thompson, Barry H ^d   Coté, Timothy R ^a  

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

^b KECK GRADUATE INSTITUTE   (United States)

^c UNIVERSITY OF MINNESOTA   (United States)

^d American College of Medical Genetics   (United States)

Author keywords

Clinical development time; Inborn errors of metabolism; Investigational new drug application; Lysosomal storage diseases; Mitochondrial diseases; Orphan drug designation

Indexed keywords

4 PHENYLBUTYRIC ACID; AGALSIDASE BETA; ALGLUCERASE; ALPHA 1 ANTITRYPSIN; AMMONUL; BENZOIC ACID; BETAINE; CARNITINE; GALSULFASE; IDURONATE 2 SULFATASE; IMIGLUCERASE; KUVAN; LARONIDASE; MERCAPTAMINE; MIGLUSTAT; NEW DRUG; NITISINONE; ORPHAN DRUG; PEGADEMASE; PHENYLACETIC ACID; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; SACROSIDASE; SAPROPTERIN; SUCRAID; TIOPRONIN; TRIENTINE; UCEPHAN; UNCLASSIFIED DRUG; ZINC ACETATE;

ACT; ARTICLE; CLINICAL DEVELOPMENT PLAN; DISORDERS OF MITOCHONDRIAL FUNCTIONS; DRUG APPROVAL; DRUG EFFICACY; DRUG INDICATION; FATTY ACID OXIDATION; FOOD AND DRUG ADMINISTRATION; HUMAN; INBORN ERROR OF METABOLISM; LYSOSOME STORAGE DISEASE; PRIORITY JOURNAL; SAFETY; STATISTICAL SIGNIFICANCE;

CHILD; CHILD, PRESCHOOL; DRUG APPROVAL; DRUG TOXICITY; DRUGS, INVESTIGATIONAL; FEMALE; FORECASTING; HUMANS; INFANT; INFANT, NEWBORN; MALE; METABOLISM, INBORN ERRORS; NEEDS ASSESSMENT; ORPHAN DRUG PRODUCTION; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 77954359837     PISSN: 00314005     EISSN: 10984275     Source Type: Journal    
DOI: 10.1542/peds.2009-3246     Document Type: Article

References (9)

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