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Clinical Trials for Glycogen Storage Disease Type II

• Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
• Clinical Study for IOPD Participants Less Than or Equal to 6 Months of Age to Evaluate Efficacy and Safety of Enzyme Replacement Therapy (ERT) With Avalglucosidase Alfa
• Rossella: A Study to Evaluate the Safety, PK, Efficacy, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
• Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
• In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
• A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD
• Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease
• Efficacy and Safety of GSK3196165 Versus Placebo and Tofacitinib in Participants With Moderately to Severely Active Rheumatoid Arthritis Who Have an Inadequate Response to Methotrexate
• ZIP Study - A Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of ATB200/AT2221 in Pediatric Subjects Aged 0 to < 18 Years With Pompe Disease
• PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPD
• Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment
• A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa
• VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease
• Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)
• 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
• First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
• Immune Modulation Therapy for Pompe Disease
• Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
• A Pilot Study of Pyridostigmine in Pompe Disease
• Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease
• A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
• Avalglucosidase Alfa Extension Study
• Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
• BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
• Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
• Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease
• Albuterol in Individuals With Late Onset Pompe Disease (LOPD)
• A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
• Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
• Drug-drug Interaction Study
• Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
• 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
• Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
• A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
• A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II
• Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease