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Date: 2014-12-04 01:13:21Pediatrics Lipid storage disorders Medical genetics Lysosomal storage disease Newborn screening Inborn error of metabolism Genetic disorder Cystic fibrosis Enzyme replacement therapy Health Medicine Rare diseases | Microsoft Word - Document1Add to Reading ListSource URL: www.hrsa.govDownload Document from Source WebsiteFile Size: 35,47 KBShare Document on Facebook |
How one patient with a rare disease coped with his genetic disorderDocID: 1r9mW - View Document | |
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What is HUNTER SYNDROME? Hunter syndrome, or mucopolysaccharidosis II (MPS II), is a rare and progressive lysosomal storage disease inherited in an X-linked recessiveDocID: 1lIxr - View Document | |
MUCOPOLYSACCHARIDOSES Rare Diseases Unit of the Finnish Association of People with Physical Disabilities Support for this guide was provided by Genzyme.DocID: 1fJSQ - View Document | |
May 27, 2014 BioMarin Doses First Patient in Phase 3 INSPIRE Trial With BMN 701 for the Treatment of Pompe Disease Proprietary Glycosylation Independent Lysosomal Targeting (GILT) Tagging Technology Has Been Shown to ImDocID: 1aiEg - View Document |