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Lysosomal storage diseases
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1	How one patient with a rare disease coped with his genetic disorder Add to Reading List Source URL: www.epp-deutschland.de Language: English - Date: 2015-04-23 11:35:57 Medicine Health Clinical medicine Rare diseases Porphyrias Lysosomal storage diseases RTT Erythropoietic protoporphyria Glycogen storage disease type II Disease
2	MUCOPOLYSACCHARIDOSES Rare Diseases Unit of the Finnish Association of People with Physical Disabilities Support for this guide was provided by Genzyme. Add to Reading List Source URL: www.invalidiliitto.fi Language: English - Date: 2015-07-19 05:13:47 Mucopolysaccharidosis Hunter syndrome Hurler syndrome Morquio syndrome Sanfilippo syndrome Maroteaux–Lamy syndrome Genetic disorder Lysosomal storage disease Sly syndrome Health Syndromes Rare diseases
3	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 Im Add to Reading List Source URL: www.apjohnventures.com Language: English - Date: 2014-05-29 15:30:33 Genetic genealogy BioMarin Pharmaceutical Glycogen storage disease type II Acid alpha-glucosidase Lysosomal storage disease Mucopolysaccharidosis Alglucosidase alfa Neuronal ceroid lipofuscinosis Tetrahydrobiopterin Health Rare diseases Medicine
4	先進国における希少疾患対策の分析－国際連携体制－ Add to Reading List Source URL: www.opencimi.org Language: English - Date: 2014-11-03 05:22:06 Abdominal pain Medical terms Rare diseases Connective tissue diseases Sarcoidosis Lysosomal storage disease Colitis Chronic Rheumatoid arthritis Health Medicine Autoimmune diseases
5	Microsoft Word - Apjohn Ventures Fund 2Q10 Letter Final Add to Reading List Source URL: www.apjohnventures.com Language: English BioMarin Pharmaceutical Rare diseases Orphan drugs Lysosomal storage diseases Glycogen storage disease type II Mucopolysaccharidosis Mannose 6-phosphate Hurler syndrome Phenylketonuria Health Medicine Genetic genealogy
6	Mechanisms in Cell Biology and Diseases – Publications ________________________________________________________________________________ Journal ArticlesKakavanos R, Lehn P, Callebaut I, Meikle PJ, Parki Add to Reading List Source URL: unisa.edu.au Language: English - Date: 2012-07-17 01:09:00 Mucopolysaccharidosis Lysosomal storage disease Sulfatase Brooks
7	PDF Document Add to Reading List Source URL: www.pbs.gov.au Language: English - Date: 2014-10-01 21:56:22 Lysosomal storage diseases Orphan drugs Glycogen storage disease type II Hepatology Alglucosidase alfa Pharmaceutical Benefits Scheme Amyotrophic lateral sclerosis Clinical trial Health Medicine Rare diseases
8	Fabry AwarenessPDF1 Add to Reading List Source URL: www.shire.com Language: English - Date: 2015-04-01 10:25:49 Fabry disease Lysosomal storage disease Medicine Fabry Genetic disorder Lipid storage disorders Rare diseases Health
9	Newborn Screening for MPS I: Final Report from the Condition Review Workgroup Alex R. Kemper, MD, MPH, MS February 13, 2015 Add to Reading List Source URL: www.hrsa.gov Language: English Rare diseases Hurler syndrome Syndromes Lysosomal storage disease Mucopolysaccharidosis Iduronidase Coarse facial features Hematopoietic stem cell transplantation Newborn screening Health Medicine Biology
10	Microsoft WordAlglucosidase Genzyme PSD March 2013 FINAL.doc Add to Reading List Source URL: www.pbs.gov.au Language: English - Date: 2014-10-01 21:56:23 Lysosomal storage diseases Glycogen storage disease type II Hepatology Alglucosidase alfa Pharmaceutical Benefits Scheme Amyotrophic lateral sclerosis Clinical trial Health Medicine Rare diseases

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