Showing 16 of 16 recruiting trials for “glycogen-storage-disease-due-to-aldolase-a-deficiency”
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
👨⚕️ Trial Transparency email recommended (Toll free for US & Canada)📍 1 site📅 Started May 2025View details ↗
RecruitingNCT06795152 ↗
Rare Glycogen Storage Diseases Natural History Study
Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
RecruitingNCT06121011 ↗
A Global Prospective Observational Registry of Patients With Pompe Disease
RecruitingNCT06214507 ↗
Danon Disease Natural History Study
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
👨⚕️ Joseph Rossano, MD, Children's Hospital of Philadelphia📍 6 sites📅 Started Sep 2023View details ↗
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
RecruitingNCT03564561 ↗
Natural History of Pompe Disease
RecruitingNCT04639336 ↗
Cognitive and Neurological Pathologies in Pompe Disease
Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
RecruitingNCT01665326 ↗
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
RecruitingNCT02399748 ↗
A Long-term Study for the Outcome of Pompe Disease
Pompe Disease Registry Protocol
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