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BioMarin Pharmaceutical
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41	FOOD AND DRUG ADMINISTRATION (FDA) Add to Reading List Source URL: www.fda.gov Language: English BioMarin Pharmaceutical Morquio syndrome Mucopolysaccharidosis Food and Drug Administration Health Medicine Pharmaceutical sciences
42	BATTELLE FUNDING: PROGRESS REPORT Add to Reading List Source URL: www.vpresearch.iastate.edu Language: English - Date: 2012-09-10 17:27:58 Mucopolysaccharidosis BioMarin Pharmaceutical Perilipin Lysosomal storage disease Rothschild Sanfilippo syndrome Animal Genetics Cattle Battelle Memorial Institute Biology Health Genetics
43	National Organization for Rare Disorders Remarks before the FDA Endocrinologic and Metabolic Drugs Advisory Committee Silver Spring, Maryland November 19, 2013 Good afternoon. My name is Diane Edquist Dorman, Vice Presid Add to Reading List Source URL: www.rarediseases.org Language: English - Date: 2013-11-22 12:36:21 Rare diseases Drug discovery Orphan drug Pharmaceuticals policy Morquio syndrome Pharmaceutical industry BioMarin Pharmaceutical Mucopolysaccharidosis National Organization for Rare Disorders Health Pharmaceutical sciences Medicine
44	October 28, 2013 Elizabeth M. Murphy, Esq. Secretary U.S. Securities and Exchange Commission 100 F Street, NE Add to Reading List Source URL: www.sec.gov Language: English - Date: 2013-10-30 11:08:49 New York City Economy of New York Stock market BioMarin Pharmaceutical Economy of the United States NASDAQ
45	Summary ICORD ACCORD workshops with ICORD’s different stakeholders ICORD Meets Industry June 18, 2014, Karolinska University Hospital, Stockholm, Sweden ICORD is a global organization, in which all stakeholders withi Add to Reading List Source URL: icord.se Language: English - Date: 2014-07-06 16:48:02 Orphan drug BioMarin Pharmaceutical Karolinska Institutet Pharmaceutical industry Karolinska University Hospital Genzyme Technology Health Epidemiology Rare disease
46	BioMarin is currently seeking patients for a Phase 3 clinical trial in Late Onset Pompe Disease. This phase 3 study incorporates a switchover design in which all patients switch directly from Myozyme® or Lumizyme® 1to Add to Reading List Source URL: www.unitedpompe.com Language: English - Date: 2014-06-16 18:19:57 Glycogen storage disease type II Hepatology BioMarin Pharmaceutical Sanofi Alglucosidase alfa Genzyme Clinical trial ClinicalTrials.gov Medicine Health Science
47	Association for Glycogen Storage Disease (UK) Issue 10 February 2003 POMPE’S Add to Reading List Source URL: www.pompe.org.uk Language: English - Date: 2007-02-15 10:12:22 Lysosomal storage diseases Glycogen storage disease type II Hepatology Lipid storage disorders Genzyme BioMarin Pharmaceutical Fabry disease Enzyme replacement therapy Clinical trial Health Medicine Rare diseases
48	Copy of Orphan Drug Designation List as of[removed]15PM).xlsx Add to Reading List Source URL: www.hrsa.gov Language: English - Date: 2013-12-06 13:38:37 Pharmacokinetics Givinostat BioMarin Pharmaceutical CSL Behring Pharmaceutical sciences Pharmacology Orphan drugs PEGylation

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