LOPD

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Trials to join now! 1

One-shot gene therapy aims to free pompe patients from lifelong infusions

Disease control Recruiting now

This 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: LOPD

Phase: PHASE1, PHASE2 • Sponsor: AskBio Inc • Aim: Disease control

Last updated May 11, 2026 20:52 UTC

LOPD

Get alerted when new LOPD trials appear

One-shot gene therapy aims to free pompe patients from lifelong infusions