LGMD2C

Clinical trials for LGMD2C explained in plain language.

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First gene therapy tested in children with rare muscle disease

Disease control Ongoing

This early-stage study is testing the safety of a single-dose gene therapy called ATA-200 in children with a rare genetic muscle disorder called limb girdle muscular dystrophy type 2C. The therapy aims to deliver a working copy of a faulty gene directly into the body via an intra…

Matched conditions: LGMD2C

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

Last updated Mar 09, 2026 14:23 UTC

LGMD2C

Get alerted when new LGMD2C trials appear

First gene therapy tested in children with rare muscle disease