LGMDR9

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Not yet finished but already full! 1

Gene therapy trial offers hope for rare muscle disease

Disease control Ongoing

This early-stage study is testing whether a new gene therapy called GNT0006 is safe for people with FKRP-related limb-girdle muscular dystrophy (LGMDR9). Six patients will receive the treatment, which delivers a working copy of the FKRP gene directly to muscle cells. Researchers …

Matched conditions: LGMDR9

Phase: PHASE1 • Sponsor: Atamyo Therapeutics • Aim: Disease control

Last updated Mar 31, 2026 12:11 UTC

LGMDR9

Get alerted when new LGMDR9 trials appear

Gene therapy trial offers hope for rare muscle disease