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Drug discovery / Pharmacology / Epidemiology / Pharmaceuticals policy / Orphan drug / Orphan Drug Act / Growth hormone treatment / Food and Drug Administration / Rare disease / Health / Medicine / Pharmaceutical sciences


What the Orphan Drug Act Has Done Lately for Children With Rare Diseases: A 10-Year Analysis Chandana Thorat, Kui Xu, Scott N. Freeman, Renan A. Bonnel, Francesca Joseph, M. Ian Phillips and Menfo A. Imoisili Pediatrics;
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Document Date: 2012-02-22 08:02:14

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City

Claremont / /

Company

Lennox / Web / /

Country

United States / /

Currency

pence / USD / /

/

Event

FDA Phase / /

Facility

MPHb aKeck Graduate Institute of Applied Life Sciences / Pub L No. / /

IndustryTerm

oncology products / Treatment of Fabry disease / biological products / Rare Disease Therapies / Treatment of Lennox-Gastaut syndrome Treatment / advocacy groups / Treatment of von Willebrand disease / patient support / online version / treatment products / Treatment of growth failure / biological product / pediatric / Noonan syndrome Treatment / pediatric products / rst product / orphan products / Treatment of Lennox-Gastaut Syndrome / Crohn disease Treatment / designated orphan products / Prader-Willi syndrome Treatment / Treatment of growth hormone insensitivity syndrome / Treatment of glycogen storage disease / orphan product / drug products / Treatment of growth hormone insensitivity syndrome Treatment / /

MedicalCondition

pediatric diseases / endocrine and metabolic disorders / Rare diseases / Elliott E. Rare childhood diseases / mucopolysaccharidosis / Krabbe disease / targeted disease / refractory acute lymphoblastic leukemia / Kawasaki disease / purpura fulminans Treatment / infantile spasms / glycogen storage disease / hematologic disorders / Maroteaux-Lamy syndrome / encephalopathy / growth hormone insensitivity syndrome / Lyme disease / such diseases / von Willebrand disease Biological N-acetylgalactosamine4 / orphan diseases / Tambuyzer E. Rare diseases / orphan disease / tyrosinemia type / Hunter syndrome / far lower median disease / hemophilia A / disease / parathyroid cancer / median disease / severe / malignant osteopetrosis Drug Biological Biological Drug Biological THORAT / rare disease / hereditary angioedema Treatment / diseases / pulmonary emboli / von Willebrand disease / ulcerative colitis Mucopolysaccharidosis / Children With Rare Diseases / thrombosis / Balsalazide disodium Ribavirin Botulism / chronic hepatitis C / /

MedicalTreatment

invasive procedures / enzyme replacement therapy / /

Organization

Rare Disorders and Genetic Alliance / MPHb aKeck Graduate Institute of Applied Life Sciences / Center for Rare Disease Therapies / US Food and Drug Administration / National Organization for Rare Disorders / FDA / American Academy of Pediatrics / American Academy of Pediatrics FINANCIAL DISCLOSURE / /

Person

Ian Phillips / David Maybee / Kui Xu / Arch Dis / M. Ian Phillips / Menfo A. Imoisili / Menfo A. Imoisili Pediatrics / Drs Freeman / Drs Timothy Cote / A. Bonnel / Patricia Dinndorf / Francesca Joseph / Kevin Shannon / Elliott E. Rare / Renan A. Bonnel / N. Freeman / Scott N. Freeman / Debra Lewis / A. Imoisili / /

Position

investigator / Commissioner / US Food and / Hunter / /

Product

Topiramate / Balsalazide disodium / Meloxicam / NeoProfen / Ribavirin / Act / United States / /

ProvinceOrState

Illinois / California / /

Region

Mediterranean / /

Technology

pdf / drug development / /

URL

www.pediatrics.org/cgi/doi/10.1542/peds.2011-1798 / http /

SocialTag