Glycogen Storage Disease Type II
Pipeline by Development Stage
On Market (1)
Approved therapies currently available
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Showing 15 of 28 trials with date data
Clinical Trials (29)
Total enrollment: 2,873 patients across 29 trials
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment
Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for 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
Immune Tolerance Induction Study
Growth and Development Study of Alglucosidase Alfa
High Dose or High Dose Frequency Study of Alglucosidase Alfa
Late-Onset Treatment Study Extension Protocol
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
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
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602
A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease
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
Avalglucosidase Alfa Extension Study
A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II
rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)
Alglucosidase Alfa Temporary Access Program
Avalglucosidase Alfa Pregnancy Study
A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
Alglucosidase Alfa Pompe Safety Sub-Registry
Pompe Lactation Sub-Registry
Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease
Pompe Disease Registry Protocol
Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease