POMPE DISEASE (LATE-ONSET)
Clinical trials for POMPE DISEASE (LATE-ONSET) explained in plain language.
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One-shot gene therapy aims to free pompe patients from lifelong infusions
Disease control Recruiting nowThis study tests a single intravenous dose of AB-1009 gene therapy in 12 adults with late-onset Pompe disease. Participants must have been on enzyme replacement therapy for at least 6 months. The goal is to see if the treatment is safe and can reduce or replace the need for regul…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE1, PHASE2 • Sponsor: AskBio Inc • Aim: Disease control
Last updated May 11, 2026 20:52 UTC
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Shocking muscles back to life: new exercise hope for ALS and SMA patients
Symptom relief Recruiting nowThis study tests a special exercise method using whole-body electrical muscle stimulation (WB-EMS) in adults with neuromuscular diseases like ALS, SMA, and muscular dystrophy. Because these conditions make normal exercise hard, WB-EMS directly triggers muscle contractions without…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: NA • Sponsor: University of Missouri-Columbia • Aim: Symptom relief
Last updated May 15, 2026 11:54 UTC
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New study to monitor pompe disease in pregnancy
Knowledge-focused Recruiting nowThis study follows pregnant women with Pompe disease to see how the condition and its treatments affect pregnancy and baby growth. About 20 women will join, and their babies will be tracked for three years after birth. No new treatments are tested; the goal is simply to gather in…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Sponsor: Genzyme, a Sanofi Company • Aim: Knowledge-focused
Last updated May 15, 2026 11:57 UTC