Condition
Pompe Disease Infantile-Onset clinical trials and research
As of November 12, 2026, Find a Trial lists 3 Pompe Disease Infantile-Onset clinical trials that are recruiting, at sites in 2 countries (most in United States (2), France (1)). By phase: Phase 1 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 3Clinical trials
- 2Countries
Newest Pompe Disease Infantile-Onset clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Using RNA analysis to improve diagnosis in patients with rare muscle diseases | — | Marseille | Recruiting | Feb 18, 2025 |
| Registry for patients with lysosomal storage diseases | — | San Francisco, California | Recruiting | Nov 17, 2022 |
| In utero enzyme replacement therapy for lysosomal storage disorders | Phase 1 | San Francisco, California | Recruiting | Aug 31, 2020 |
All recruiting trials (3)
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Using RNA analysis to improve diagnosis in patients with rare muscle diseases
This study is testing if analyzing RNA from muscle samples can help doctors better diagnose patients with rare muscle diseases who have previously received negative DNA test results.
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Registry for patients with lysosomal storage diseases
This study is collecting information from patients with lysosomal storage diseases to see how these conditions progress and how treatments can improve care for them and their families.
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In utero enzyme replacement therapy for lysosomal storage disorders
This study is testing whether giving enzyme replacement therapy to unborn babies with lysosomal storage disorders can improve their health and development before they are born.
Related topics
Quick answers
How many Pompe Disease Infantile-Onset clinical trials are recruiting right now?
3, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 2 countries. The most sites are in United States (2 trials), France (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 1: 1. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.