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Myopathy caused by variation in FKTN

MONDO:0700067

Any myopathy in which the cause of the disease is a variation in the FKTN gene.

Also known as: FKTN myopathy, FKTN-related myopathy, myopathy caused by mutation in FKTN

4 clinical trials for this condition and its sub-types, 1 tagged with Myopathy caused by variation in FKTN itself.

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Where it sits in the disease tree

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Part of

↑ Myopathy (990) ↑ Hereditary skeletal muscle disorder (406) ↑ Qualitative or quantitative defects of protein involved in O-glycosylation of alpha-dystroglycan (8)

Sub-types of Myopathy caused by variation in FKTN

Including sub-types (4) Tagged with Myopathy caused by variation in FKTN (1)

Not yet finished but already full! 1

Gene therapy trial offers hope for rare muscle disease

Disease control Ongoing

This study tests a single dose of a gene therapy called AB-1003 in 10 adults with a rare genetic muscle disease (LGMD2I/R9). The goal is to see if it is safe and can help improve muscle function. Participants must be able to walk or run 10 meters in under 30 seconds.

Phase 1/2 • Sponsor: AskBio Inc • Aim: Disease control

Last updated Aug 28, 2026 00:00 UTC