Records containing: Pompe Disease
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2018-02-06A Phase 1/2 Double-Blind Study of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy
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CLINICAL_TRIALS study record update · 2017-12-08Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy
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CLINICAL_TRIALS study record update · 2017-11-17Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients With Pompe Disease
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CLINICAL_TRIALS study record update · 2017-10-23A Clinical Investigation of the Safety and Efficacy of Clenbuterol on Motor Function in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
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CLINICAL_TRIALS study record update · 2017-07-18A Long-term Study for the Outcome of Pompe Disease
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CLINICAL_TRIALS study record update · 2017-04-04ClinicalTrials.gov study NCT02120235
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CLINICAL_TRIALS study record update · 2017-03-20Search for Serum/Plasma Biomarkers in Pompe's Disease
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CLINICAL_TRIALS study record update · 2016-09-28Pregnancy and Birth Outcome in Women With Pompe Disease
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CLINICAL_TRIALS study record update · 2016-09-20Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
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CLINICAL_TRIALS study record update · 2016-04-15Immune Modulation Therapy for ERT-naïve or ERT-treated Pompe Disease Patients
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CLINICAL_TRIALS study record update · 2015-10-27A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale
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CLINICAL_TRIALS study record update · 2015-05-05Screening Protocol to Evaluate Acid Alpha Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease for Potential Inclusion in Future Clinical Studies With Myozyme (Alglucosidase Alfa)
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CLINICAL_TRIALS study record update · 2015-04-01Efficacy of Continuous Positive Airway Pressure of Treatment of Hypernasality of Children With Infantile Pompe Disease
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CLINICAL_TRIALS study record update · 2015-03-24Muscle Response to Enzyme Replacement Therapy in Pompe Disease
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CLINICAL_TRIALS study record update · 2014-11-13A Prospective Multinational, Multicenter, Clinical Trial of the Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) in Cross-Reacting Immunologic Material-Positive Patients With Classical Infantile Pompe Disease
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CLINICAL_TRIALS study record update · 2014-05-13An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme®-Naive, CRIM(-) Patients With Infantile-onset Pompe Disease
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CLINICAL_TRIALS study record update · 2014-02-05Open-Label, Pilot Study of the Safety, Pharmacokinetics and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase (rhGAA) as Enzyme Replacement Therapy in Siblings With Glycogen Storage Disease Type II (GSD-II).
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CLINICAL_TRIALS study record update · 2014-02-05An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-II
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CLINICAL_TRIALS study record update · 2013-12-04A Long Term Follow up Study in Late-onset Pompe Disease
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CLINICAL_TRIALS study record update · 2013-08-30A Natural History Study of Adult Onset Pompe Disease Using Muscle MRI
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CLINICAL_TRIALS study record update · 2013-01-01Newborn Screening Assay of Pompe's Disease
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CLINICAL_TRIALS study record update · 2011-12-01Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia
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CLINICAL_TRIALS study record update · 2011-08-04Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry
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CLINICAL_TRIALS study record update · 2011-04-22An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Alglucosidase Alfa Treatment in Patients With Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen
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CLINICAL_TRIALS study record update · 2008-06-05A Multicenter Study to Evaluate and Characterize the Effects of Pharmacological Chaperones in Cell Lines Derived From Blood and Skin Samples From Patients With Pompe Disease