WORKSHOP SUMMARY Sponsored by: Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA) Office of the Commissioner, Office of Orphan Products Development (OOPD), FDA National Institutes of Healt - Rare Diseases Clinical Research Network - Document - PDFSEARCH.IO - Document Search Engine

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	WORKSHOP SUMMARY Sponsored by: Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA) Office of the Commissioner, Office of Orphan Products Development (OOPD), FDA National Institutes of Healt Add to Reading List Document Date: 2012-06-15 15:38:24 Open Document File Size: 1,20 MB Share Result on Facebook City Dr. Groft / la Paz / Washington / DC / / Company National Academies Press / AVI BioPharma / Genzyme / United BioSource Corporation / / Event Person Travel / FDA Phase / / Facility NIH National Institute of Neurological Disorders / Eunice Kennedy Shriver National Institute of Child Health / Institute of Medicine / NIH Eunice Kennedy Shriver National Institute of Child Health / Children’s Hospital / Clinical Center / University of California Davis / Children’s National Medical Center / University of Iowa / Massachusetts General Hospital / National Human Genome Research Institute / University of South Florida / Children’s Hospital of Pittsburgh / National Institute of Neurological Disorders / University of Minnesota / Duke University Medical Center / / IndustryTerm control / magnetic resonance imaging patterns / biotechnology / important tool / placebo / study protocols / Therapeutics for Rare and Neglected Diseases / / MedicalCondition why rare disease / rare diseases / infantile Pompe disease / Urea Cycle Disorders / individual disorders / Brain Disease / Eosinophilic Esophagitis / Fabry Disease / Rare Disorders / diseases / 5q spinal muscular atrophy / eosinophilic esophagitis using / Muscular Dystrophy / Krabbe disease / Rare Genetic Diseases / Huntington’s Disease / Parent Project Muscular Dystrophy / common diseases / Pompe Disease / disease / Mucopolysaccharidosis IIIA / given rare disease / Neglected Diseases / rare disease / several rare diseases / Rare and Neglected Diseases / GM2 Gangliosidosis / Globoid Cell Leukodystrophy / rare neurodegenerative genetic diseases / measure disease / / Organization National Human Genome Research Institute / office of New Drugs / NIH National Institute of Neurological Disorders / NIH Clinical Center / National Center / FDA’s office of Orphan Products Development / National Institute of Neurological Disorders and Stroke / SMA Foundation / National Center for Advancing Translational Sciences / Children’s Hospital of Pittsburgh of UPMC / University of Iowa / FDA’s OOPD / National Institute of Health / University of South Florida / Food and Drug Administration / Children’s National Medical Center / Institute of Medicine / Massachusetts General Hospital / Children’s Hospital Boston / Congress / NIH office of Rare Diseases Research / University of California Davis / Clinical Center Office / National Institute of Child Health / office of Orphan Products Development / Clinical Center / Center for Drug Evaluation / University of Minnesota / Duke University Medical Center / office of Rare Disease Research / / Person Florian Eichler / Annette Stemhagen / Gayatri Rao / Jeffrey Krischer / Priya Kishnani / Christopher Austin / Marc Walton / Edward Kaye / Lawrence Charnas / Richard Moscicki / Karen Chen / Jane Paulsen / Retrospective Chart Reviews Robert Fiorentino / Meral Gunay-Aygun / Nuria Carrillo / Patricia Furlong / David Pearce / Stephen C. Groft / Craig McDonald / Marshall Summar / Moving Forward John McKew / Basil Darras / P.K. Tandon / John Gallin / Elsa Shapiro / Patrick Haslett / Maria Escolar / / Position Summary Executive / Acting Director / Director for Rare Diseases / good data coordinator / Director / Scientific Director / Division of Pre-Clinical Innovation / Commissioner / / Product Myozyme / Fabrazyme / pre-Investigational New Drug / Fabrazyme® (agalsidase beta) / / ProvinceOrState Minnesota / Iowa / / PublishedMedium the Natural History / / Region South Florida / / Technology genotyping / Human Genome / biomaterials / magnetic resonance imaging / genotyping technology / Biotechnology / study protocols / gene delivery / drug development / / SocialTag National Institutes of Health Rare diseases Pharmacology Nursing research Epidemiology Clinical trial Niemann–Pick disease Rare Diseases Clinical Research Network Biomarker Health