Pompe disease
Clinical trials for Pompe disease explained in plain language.
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Can an oral pill ease breathing and muscle weakness in pompe disease?
Disease control Not yet recruitingThis early-stage trial is testing an experimental oral medication called S-606001 in adults with late-onset Pompe disease, a condition that causes progressive muscle weakness and breathing problems. The main goal is to see if the drug is safe and tolerable. Researchers will also …
Matched conditions: POMPE DISEASE
Phase 1 • Sponsor: Shionogi • Aim: Disease control
Last updated Aug 28, 2026 00:00 UTC
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Can an oral pill boost muscle strength in pompe disease?
Disease control Not yet recruitingThis study tests the long-term safety and effectiveness of an experimental oral drug called S-606001 in adults with late-onset Pompe disease, a genetic condition that causes progressive muscle weakness. Participants who completed a prior S-606001 study can join, and they will rec…
Matched conditions: POMPE DISEASE
Phase 2 • Sponsor: Shionogi • Aim: Disease control
Last updated Aug 08, 2026 00:03 UTC