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| First Page | Document Content | |
|---|---|---|
 Medicine Finnish heritage disease Compound heterozygosity Lysosomal storage disease Neuronal ceroid lipofuscinosis Congenital chloride diarrhea Linkage disequilibrium Congenital nephrotic syndrome CLN3 Health Rare diseases Biology |
| How one patient with a rare disease coped with his genetic disorderDocID: 1r9mW - View Document |
| CrossMark_Color_flat+clickDocID: 1r0lC - View Document |
| 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 |