LATE-ONSET POMPE DISEASE
Clinical trials for LATE-ONSET POMPE DISEASE explained in plain language.
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New drug DNL952 enters human testing for pompe disease
Disease control Recruiting nowThis early-stage trial is testing a new drug called DNL952 in 32 adults with late-onset Pompe disease. The main goal is to check if the drug is safe and how the body processes it. Participants will receive the drug through an IV infusion at different doses.
Matched conditions: LATE-ONSET POMPE DISEASE
Phase: PHASE1 • Sponsor: Denali Therapeutics Inc. • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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New pill could boost breathing in pompe disease – early trial underway
Disease control Recruiting nowThis study tests an experimental oral drug called S-606001 in 45 adults with late-onset Pompe disease. Participants take the drug or a placebo on top of their standard enzyme replacement therapy. The main goal is to see if the drug improves lung function and walking ability over …
Matched conditions: LATE-ONSET POMPE DISEASE
Phase: PHASE2 • Sponsor: Shionogi • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Pompe disease patients invited to join worldwide registry
Knowledge-focused Recruiting nowThis study is a global registry that will follow about 500 people with Pompe disease over time. It includes both treated and untreated patients, and aims to collect real-world data on treatment safety, effectiveness, and quality of life. No new treatments are being tested—this is…
Matched conditions: LATE-ONSET POMPE DISEASE
Sponsor: Amicus Therapeutics • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:00 UTC
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Major study aims to better measure muscular dystrophy progression
Knowledge-focused Recruiting nowThis 24-month observational study will follow up to 1000 people with certain types of muscular dystrophy (LGMD, DM2, and late-onset Pompe disease) aged 6-50. Researchers want to see if specific physical tests, like the North Star Assessment and a 100-meter walk, are good ways to …
Matched conditions: LATE-ONSET POMPE DISEASE
Sponsor: Virginia Commonwealth University • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:57 UTC