Glycogen Storage Disease Type Ii
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-03-23Clinical Trial Update: China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD [Phase 4]
Phase 4 post-approval commitment study of avalglucosidase alfa for Pompe disease, currently recruiting in China in participants with infanti…
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CLINICAL_TRIALS trial_result · 2023-11-14Clinical Trial Update: Nutritional Therapy in Late-onset Pompe Disease [Phase 2]
Phase 2 trial evaluating nutritional therapy for late-onset Pompe disease with muscle loss; current status is unknown according to ClinicalT…
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CLINICAL_TRIALS trial_result · 2022-08-26Clinical Trial Update: Growth and Development Study of Alglucosidase Alfa [Phase 4]
A Phase 4 clinical trial update for alglucosidase alfa investigating growth and development in Pompe disease, with results posted after comp…
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CLINICAL_TRIALS trial_result · 2022-04-13Clinical Trial Update: Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment [Phase 4]
A Phase 4 trial updating efficacy and safety of one-year alglucosidase alfa treatment in Chinese patients with infantile-onset Pompe disease…
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CLINICAL_TRIALS trial_result · 2021-06-11Clinical Trial Update: Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease [Phase 4]
Phase 4 pharmacokinetic study of alglucosidase alfa in Pompe disease patients has completed and results were posted in 2021.
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CLINICAL_TRIALS trial_result · 2021-04-08Clinical Trial Update: Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease [Phase 3]
Phase 3 trial comparing avalglucosidase alfa to alglucosidase alfa in late-onset Pompe disease patients who have not been treated before, wi…
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CLINICAL_TRIALS trial_result · 2020-10-12Clinical Trial Update: Immune Tolerance Induction Study [Phase 4]
Phase 4 trial update for Immune Tolerance Induction in Pompe disease was terminated with results posted in 2020.
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CLINICAL_TRIALS trial_result · 2017-05-16Clinical Trial Update: Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe Disease
This completed clinical trial update investigates using cardiopulmonary exercise testing to quantify the response to enzyme replacement ther…
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CLINICAL_TRIALS trial_result · 2016-01-18Clinical Trial Update: A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease [Phase 4]
Phase 4 noninferiority trial evaluating alglucosidase alfa manufactured at 160 L vs 4000 L scales in treatment-naïve infants with Pompe dise…
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CLINICAL_TRIALS trial_result · 2015-05-05Clinical Trial Update: A Prospective, Observational Study in Patients With Late-Onset Pompe Disease
This clinical trial update notes the completion of a prospective, observational study in patients with late-onset Pompe disease (Glycogen St…
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CLINICAL_TRIALS trial_result · 2014-12-02Clinical Trial Update: Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa [Phase 4]
Phase 4 exploratory study assessing muscle biopsy changes in patients with late-onset Pompe disease treated with alglucosidase alfa; results…
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CLINICAL_TRIALS trial_result · 2014-11-13Clinical Trial Update: Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease [Phase 2]
Phase 2 clinical trial assessing the safety and efficacy of recombinant human acid alpha-glucosidase for classical infantile Pompe disease; …
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CLINICAL_TRIALS trial_result · 2014-05-13Clinical Trial Update: An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease [Phase 4]
Phase 4 exploratory trial evaluating the safety and efficacy of prophylactic immunomodulatory treatment in Myozyme-naive CRIM-negative infan…
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CLINICAL_TRIALS trial_result · 2014-02-06Clinical Trial Update: Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®
This clinical trial update describes the progression of a patient with severe late-onset Pompe disease and pulmonary dysfunction who is bein…
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CLINICAL_TRIALS trial_result · 2014-02-05Clinical Trial Update: Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support [Phase 3]
A Phase 3 trial evaluating rhGAA (alglucosidase alfa) in patients with advanced late-onset Pompe disease on respiratory support has complete…
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CLINICAL_TRIALS trial_result · 2014-02-05Clinical Trial Update: A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II [Phase 2]
Phase 2 study evaluating the safety and pharmacokinetics of recombinant human acid alpha-glucosidase (rhGAA) in siblings with glycogen stora…
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CLINICAL_TRIALS trial_result · 2014-02-05Clinical Trial Update: rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) [Phase 1, Phase 2]
A completed Phase 1/2 trial evaluating rhGAA (alglucosidase alfa) in infants with Glycogen Storage Disease Type II (Pompe disease).
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CLINICAL_TRIALS trial_result · 2014-02-05Clinical Trial Update: Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies [Phase 2]
An extension study assessing long-term safety and efficacy of Myozyme (alglucosidase alfa) in Pompe disease patients who were previously enr…
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CLINICAL_TRIALS trial_result · 2012-01-05Clinical Trial Update: Late-Onset Treatment Study Extension Protocol [Phase 4]
Phase 4 study extension for late-onset Pompe disease and glycogen storage disease type II; the trial is completed and results were posted in…