What the orphan drug act has done lately for children with rare diseases: A 10-year analysis

Pediatrics

Volumn 129, Issue 3, 2012, Pages 516-521

  Thorat, Chandana ^a   Xu, Kui ^b   Freeman, Scott N ^b   Bonnel, Renan A ^b   Joseph, Francesca ^b   Phillips, M Ian ^a   Imoisili, Menfo A ^b  

^a KECK GRADUATE INSTITUTE   (United States)

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

Author keywords

Orphan Drug Act; Orphan drugs; Pediatrics; Rare diseases

Indexed keywords

ADALIMUMAB; ALPHA GALACTOSIDASE; BENZOIC ACID; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 PLUS VON WILLEBRAND FACTOR; CERAMIDE TRIHEXOSIDE; COLCHICINE; COMPLEMENT COMPONENT C1S INHIBITOR; FIBRINOGEN; GROWTH HORMONE; HISTRELIN; IDURONATE 2 SULFATASE; INFLIXIMAB; LARONIDASE; MECASERMIN RINFABATE; MELOXICAM; N ACETYLGALACTOSAMINE 4 SULFATASE; NITISINONE; ORPHAN DRUG; PHENYLACETIC ACID; PROTEIN C; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; RECOMBINANT SOMATOMEDIN C; RILONACEPT; RUFINAMIDE; SAPROPTERIN; TOPIRAMATE; UNINDEXED DRUG; VIGABATRIN;

ANGIONEUROTIC EDEMA; ARTICLE; BLEEDING; BRAIN DISEASE; CHILD CARE; CROHN DISEASE; DATA BASE; DRUG APPROVAL; DRUG LEGISLATION; DRUG MARKETING; FABRY DISEASE; FAMILIAL MEDITERRANEAN FEVER; FIBRINOGEN DEFECT; GLYCOGEN STORAGE DISEASE TYPE 2; GROWTH DISORDER; GROWTH HORMONE DEFICIENCY; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; HYPERAMMONEMIA; HYPERPHENYLALANINEMIA; INFANTILE SPASM; JUVENILE RHEUMATOID ARTHRITIS; LENNOX GASTAUT SYNDROME; MAROTEAUX LAMY SYNDROME; ONSET AGE; PRECOCIOUS PUBERTY; PREVALENCE; PRIORITY JOURNAL; PROTEIN C DEFICIENCY; RARE DISEASE; SHORT STATURE; THROMBOSIS; TYROSINEMIA; ULCERATIVE COLITIS; VON WILLEBRAND DISEASE;

CHILD; CHILD WELFARE; CHILD, PRESCHOOL; DATABASES, FACTUAL; DRUG APPROVAL; FEMALE; HUMANS; INFANT; MALE; MARKETING; ORPHAN DRUG PRODUCTION; RARE DISEASES; TIME FACTORS; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84857948904     PISSN: 00314005     EISSN: 10984275     Source Type: Journal    
DOI: 10.1542/peds.2011-1798     Document Type: Article

References (11)

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