Glycogen storage disease type II

Results: 169



#Item
21Krabbe disease / Lysosomal storage disease / Glycogen storage disease type II / Galactosylceramidase / Newborn screening / Association of Public Health Laboratories / Acid alpha-glucosidase / Centers for Disease Control and Prevention / Health / Rare diseases / Medicine

Lysosomal Storage Disorder Quarterly Report PROFICIENCY TESTING Volume 3, No. 4

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Source URL: www.cdc.gov

Language: English - Date: 2014-11-19 11:17:57
22Glycogen storage disease type II / Hepatology / Alglucosidase alfa / Hypersensitivity / Anaphylaxis / Medicine / Health / Allergology

HIGHLIGHTS OF PRESCRIBING INFORMATION These highlights do not include all the information needed to use LUMIZYME safely and effectively. See full prescribing information for LUMIZYME. LUMIZYME® (alglucosidase alfa), fo

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Source URL: www.accessdata.fda.gov

Language: English - Date: 2014-08-04 14:23:48
23Lysosomal storage diseases / Glycogen storage disease type II / Hepatology / Durhane Wong-Rieger / Orphan drug / Orphan Drug Act / Neglected diseases / Rieger / Health / Medicine / Rare diseases

PRESS RELEASE Contact: Brad Crittenden President, Canadian Association of Pompe Email: [removed] For Immediate Release

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Source URL: www.raredisorders.ca

Language: English
24Canadian Organization for Rare Disorders / Morquio syndrome / Orphan drug / Lysosomal storage disease / European Organization for Rare Diseases / Fabry disease / Rare Disease Day / Glycogen storage disease type II / Mental disorder / Health / Medicine / Rare diseases

The LINK Canadian Organization for

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Source URL: www.raredisorders.ca

Language: English - Date: 2009-04-01 16:11:55
25Rare diseases / Fabry disease / Lysosomal storage diseases / Renal function / Biomarker / Enzyme replacement therapy / Clinical trial / ERT / Glycogen storage disease type II / Medicine / Health / Clinical research

Amicus Therapeutics Announces Second Quarter 2007 Financial Results

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Source URL: www.fabry.org

Language: English - Date: 2014-12-02 16:03:24
26Rare diseases / Ofatumumab / Carglumic acid / Pulmonary hypertension / Glycogen storage disease type II / Orphanet / Acute myeloid leukemia / Leukemia / Medicine / Health / Hepatology

Octobre[removed]Listes des médicaments pour les maladies rares en Europe*

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Source URL: www.orpha.net

Language: English - Date: 2014-11-03 09:45:01
27Medical tests / Medical terms / Prevention / Screening / Newborn screening / Glycogen storage disease type II / Medicine / Health / Rare diseases

Users’ Guide to the SACHDNC Decision Matrix Alex R. Kemper, MD, MPH, MS January 31, 2013 Background

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Source URL: www.hrsa.gov

Language: English - Date: 2014-12-04 01:12:59
28Glycogen storage disease type II / Lysosomal storage disease / Acid alpha-glucosidase / Newborn screening / Screening / Medical genetics / Medicine / Health / Rare diseases

EVIDENCE REVIEW: POMPE DISEASE Prepared for: THE ADVISORY COMMITTEE ON HERITABLE DISORDERS IN NEWBORNS AND CHILDREN FINAL V[removed]

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Source URL: www.hrsa.gov

Language: English - Date: 2014-12-04 01:12:52
29Genetic genealogy / Rare diseases / Hepatology / Genetics / Glycogen storage disease type II / Glycogen storage disease type V / Lysosomal storage disease / Genotype / Acid alpha-glucosidase / Health / Inborn errors of carbohydrate metabolism / Medicine

ARTICLE The angiotensin-converting enzyme insertion/deletion polymorphism modifies the clinical outcome in patients with Pompe disease Paola de Filippi, PhD1, Sabrina Ravaglia, MD, PhD2, Bruno Bembi, MD3, Alfredo Costa,

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Source URL: www.nature.com

Language: English - Date: 2012-01-04 14:12:58
30Epidemiology / Glycogen storage disease type II / Hepatology / Newborn screening / Alternative medicine / University of Massachusetts Medical School / Screening / Health / Medicine / Rare diseases

Newborn Screening for Pompe Disease: Condition Review Update Alex R. Kemper, MD, MPH, MS September 13, 2012

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Source URL: www.hrsa.gov

Language: English - Date: 2014-12-04 01:13:41
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