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 Date: 2012-01-07 00:54:21Glycogen storage disease type II Lysosomal storage disease Acid alpha-glucosidase Newborn screening Screening Medical genetics Medicine Health Rare diseases |
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EVIDENCE REVIEW: POMPE DISEASE | 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 |