LOPD

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Trials to join now! 1

One-Shot gene therapy trial offers new hope for debilitating muscle disease

Disease control Recruiting now

This early-stage study is testing a single infusion of an experimental gene therapy called AB-1009 in adults with late-onset Pompe disease. The goal is to see if this one-time treatment is safe and if it can help control the disease, which causes progressive muscle weakness. Part…

Matched conditions: LOPD

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

Last updated Apr 02, 2026 03:56 UTC

LOPD

Get alerted when new LOPD trials appear

One-Shot gene therapy trial offers new hope for debilitating muscle disease