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Glycogen storage disease type II / Hepatology / Orphan drugs / Lysosomal storage disease / Enzyme replacement therapy / ERT / Alglucosidase alfa / End point of clinical trials / Amyotrophic lateral sclerosis / Health / Medicine / Rare diseases


Evidence for decline in the incidence of cystic fibrosis: a 35-year observational study in Brittany, France
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Document Date: 2013-05-26 18:16:26

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City

Leslie / Notermans / Boston / New York / Rotterdam / Cambridge / Horwitz / Bauer / Cary / Chicago / Florence / /

Company

SPSS Inc. / Marloes LC / Vulto AG / Dutch TI Pharma / SAS Institute Inc. / Erasmus MC / GE / Google / Genzyme Corporation / McGraw-Hill / Winkel LP / Cox / BioMed Central Ltd. / Creative Commons / conducted using SPSS / /

Continent

Europe / /

Country

Germany / Netherlands / United States / Canada / Australia / United Kingdom / /

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Event

FDA Phase / Business Partnership / /

Facility

Boston University / Erasmus MC University Medical Center / Sophia Children’s Hospital / Harvard Clinical Research Institute / /

IndustryTerm

treatment for an inherited muscular disorder / treatment of this disease / treatment of Pompe disease / therapy for Pompe disease / /

MedicalCondition

classic infantile Pompe disease present / Rare Diseases / Glycogen storage disease type II / pulmonary / Glycogen storage disease / Pompe disease express / Lysosomal storage disease / inherited muscular disorder / rare metabolic myopathy / MS / Pompe disease / glycogenosis type II / disease / Metabolic Diseases / respiratory failure / median disease / dysphagia / pulmonary function / alfa Background Pompe disease / classic infantile Pompe disease / glycogenosis / speech disorders / diseases / Lysosomal Storage Diseases / Acid maltase deficiency / respiratory insufficiency / deficiency / significant / progressive hypertrophic cardiomyopathy / /

MedicalTreatment

enzyme replacement therapy / /

MusicGroup

Immortal / /

OperatingSystem

Microsoft Windows / /

Organization

Harvard / Central Committee on Research Involving Human Subjects / International Pompe Association / Center for Lysosomal / Boston University / Medical Ethics Committee / Department of Paediatrics / /

Person

Marsha Zimmerman / Tim Stentiford / Guy Ashford-Smith / Helen Walker / Allan Muir / Ria Broekgaarden / Van Doorn / Thomas Schaller / Van den Hout / Rineke Nelisse / Joseph Massaro / Beatrix Fonds / Sophia Children / /

Position

author / treating physician / representative / Coo / /

Product

Sangean SR3 Radio / ERT / Europe / /

ProgrammingLanguage

ML / /

ProvinceOrState

North Dakota / Pennsylvania / Mississippi / North Carolina / /

Technology

alpha / research protocols / Drug Development / /

URL

www.biomedcentral.com/submit / http /

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