🧪 UPDATED DAILY FROM CLINICALTRIALS.GOVSynced Apr 1

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20+ recruiting trials across all rare diseases. Filter by phase, location, and condition.

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Showing 20 of 20 recruiting trials for “muscular-glycogenosis”

NARecruitingNCT07478172 ↗

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

🏥 University of Missouri-Columbia📍 1 site📅 Started Mar 2026View details ↗
RecruitingNCT07459582 ↗

Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease

👨‍⚕️ Malaya Mount, MS, RD, CDN, Connecticut Children's📍 1 site📅 Started Mar 2026View details ↗
Phase 1, PHASE2RecruitingNCT07282847 ↗

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)

🏥 AskBio Inc📍 10 sites📅 Started Feb 2026View details ↗
Phase 2RecruitingNCT07123155 ↗

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

🏥 Shionogi📍 28 sites📅 Started Oct 2025View details ↗
Phase 4RecruitingNCT06666413 ↗

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 ↗
NARecruitingNCT06852612 ↗

Dietary Treatment Strategies and Metabolic Control in Glycogen Storage Disease Type I

👨‍⚕️ Michel Hochuli, MD, PhD, Department of Diabetes, Endocrinology, Nutritional Medicine and Metabolism📍 1 site📅 Started Apr 2025View details ↗
RecruitingNCT06843330 ↗

Accuracy of Lactate Meter in GSDIa

🏥 Connecticut Children's Medical Center📍 1 site📅 Started Apr 2025View details ↗
RecruitingNCT06795152 ↗

Rare Glycogen Storage Diseases Natural History Study

👨‍⚕️ Priya Kishnani, M.D., Duke📍 1 site📅 Started Dec 2024View details ↗
Phase 1, PHASE2RecruitingNCT06735755 ↗

A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)

🏥 Beam Therapeutics Inc.📍 3 sites📅 Started Dec 2024View details ↗
RecruitingNCT06636383 ↗

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

👨‍⚕️ Medical Director, Ultragenyx Pharmaceuticals Inc.📍 19 sites📅 Started Nov 2024View details ↗
RecruitingNCT06396546 ↗

'Glycogen Storage Diseases (GSDs) in Indian Children- Establishing an Indian GSD (I-GSD) Registry'

👨‍⚕️ Dr Vikrant Sood, DM, Institute of Liver & Biliary Sciences📍 3 sites📅 Started May 2024View details ↗
Phase 1, PHASE2RecruitingNCT06391736 ↗

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

👨‍⚕️ Guang Yang, Chinese PLA General Hospital📍 1 site📅 Started Apr 2024View details ↗
RecruitingNCT06121011 ↗

A Global Prospective Observational Registry of Patients With Pompe Disease

🏥 Amicus Therapeutics📍 41 sites📅 Started Feb 2024View details ↗
Phase 3RecruitingNCT04808505 ↗

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

🏥 Amicus Therapeutics📍 14 sites📅 Started Jul 2023View details ↗
RecruitingNCT04454216 ↗

GSD VI and GSD IX Natural History

👨‍⚕️ Priya Kishnani, MD, Duke University📍 1 site📅 Started Sep 2020View details ↗
RecruitingNCT03564561 ↗

Natural History of Pompe Disease

👨‍⚕️ Helge Amthor, MD, PhD, Hôpital Raymond Poincaré📍 1 site📅 Started Jun 2019View details ↗
RecruitingNCT04639336 ↗

Cognitive and Neurological Pathologies in Pompe Disease

👨‍⚕️ Priya Kishnani, MD, Duke University📍 1 site📅 Started Aug 2016View details ↗
RecruitingNCT01665326 ↗

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

👨‍⚕️ Priya S Kishnani, MD, Duke University📍 1 site📅 Started Sep 2009View details ↗
RecruitingNCT02399748 ↗

A Long-term Study for the Outcome of Pompe Disease

👨‍⚕️ Yin-Hsiu Chien, National Taiwan University Hospital📍 1 site📅 Started Apr 2007View details ↗
NARecruitingNCT00231400 ↗

Pompe Disease Registry Protocol

👨‍⚕️ Study Director, Genzyme, a Sanofi Company📍 20 sites📅 Started Sep 2004View details ↗

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