FKRP MUTATION

Clinical trials for FKRP MUTATION explained in plain language.

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Trials to join now! 1

First human test of gene therapy for rare muscle disease begins

Disease control Recruiting now

This study is testing the safety of a single-dose gene therapy called AB-1003 in adults with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle disorder. Researchers will give increasing doses to small groups of participants to check for side effects. The goal is to…

Matched conditions: FKRP MUTATION

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

Last updated Mar 30, 2026 14:28 UTC

FKRP MUTATION

Get alerted when new FKRP MUTATION trials appear

First human test of gene therapy for rare muscle disease begins