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 · 2023-03-23An Open-label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in naïve and Alglucosidase Alfa Treated Late-onset Pompe Disease Patients.
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CLINICAL_TRIALS study record update · 2023-03-22Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle Weakness
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CLINICAL_TRIALS study record update · 2022-08-26A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa
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CLINICAL_TRIALS study record update · 2022-05-19Developing a Potential Cure for Pompe Disease: Clinical Specimen Collection From Individuals With Pompe Disease
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CLINICAL_TRIALS study record update · 2022-04-05Evaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients With Late-Onset Pompe Disease (LOPD)
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CLINICAL_TRIALS study record update · 2021-10-29Frequency of Pompe Disease in Patients Followed at CERCA for Myalgia With or Without Hyper Ckemia
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CLINICAL_TRIALS study record update · 2021-10-28Developmental Outcomes of School-aged Children With Infantile-onset Pompe Disease: A Telemedicine Approach to Assessment and Cognitive Training
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CLINICAL_TRIALS study record update · 2021-07-13A Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune Complex Mediated Reactions With Alglucosidase Alfa Treatment
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CLINICAL_TRIALS study record update · 2021-06-11A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
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CLINICAL_TRIALS study record update · 2021-05-14A Pilot Study of Diet and Exercise Therapy in Pompe Disease
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CLINICAL_TRIALS study record update · 2021-04-08The G.R.I.T. Study: Getting Global Rare Disease Insights Through Technology
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CLINICAL_TRIALS study record update · 2021-02-10Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months
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CLINICAL_TRIALS study record update · 2020-10-12An Exploratory Study of the Safety and Efficacy of Immune Tolerance Induction (ITI) in Patients With Pompe Disease Who Have Previously Received Myozyme
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CLINICAL_TRIALS study record update · 2020-09-25Electrical Impedance Myography and Muscle Ultrasound: Longitudinal Measures in Pompe Disease
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CLINICAL_TRIALS study record update · 2020-06-02A Three-Month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Versus Myozyme®/Lumizyme® in Patients With Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment With VAL-1221 in All Patients
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CLINICAL_TRIALS study record update · 2019-09-26Safety and Effectiveness of Resistance Exercise Training in Patients With Late Onset Pompe Disease- A Pilot Study
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CLINICAL_TRIALS study record update · 2019-07-05A Clinical Investigation of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease, Whether or Not Receiving Enzyme Replacement Therapy
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CLINICAL_TRIALS study record update · 2019-02-25Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices
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CLINICAL_TRIALS study record update · 2018-12-05A Pilot Study of the Effects of Oral Administration of Zavesca® on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy
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CLINICAL_TRIALS study record update · 2018-08-17An Open-Label, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Three Dosing Regimens of Oral AT2220 in Patients With Pompe Disease
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CLINICAL_TRIALS study record update · 2018-08-17An Open-Label, Multi-Center, International Study to Investigate Drug-Drug Interactions Between AT2220 and Alglucosidase Alfa in Patients With Pompe Disease
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CLINICAL_TRIALS study record update · 2018-06-11A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease
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CLINICAL_TRIALS study record update · 2018-05-22A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study
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CLINICAL_TRIALS study record update · 2018-05-15Evaluation of Respiratory and Skeletal Muscle Functions in Response to Acetylcholinesterase Inhibitors in Pompe Disease
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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