Centronuclear myopathy

MONDO:0018947

Centronuclear myopathy (CNM) is an inherited neuromuscular disorder characterized by clinical features of a congenital myopathy and centrally placed nuclei on muscle biopsy.

Also known as: CNM, centronuclear myopathy, myopathy, centronuclear, myopathy, myotubular

9 clinical trials for this condition and its sub-types, 2 tagged with Centronuclear myopathy itself.

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Where it sits in the disease tree

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↑ Congenital myopathy (75)

Sub-types of Centronuclear myopathy

X-linked myotubular myopathy 4 trials
2 sub-types
- X-linked myotubular myopathy-abnormal genitalia syndrome 0 trials
- Symptomatic form of X-linked centronuclear myopathy in female carriers 0 trials
Autosomal dominant centronuclear myopathy 0 trials
Autosomal recessive centronuclear myopathy 0 trials
2 sub-types
- Myopathy, centronuclear, 2 0 trials
- Myopathy, centronuclear, 5 0 trials
Congenital myopathy with internal nuclei and atypical cores 0 trials
Myopathy, centronuclear, 6, with fiber-type disproportion 0 trials

Including sub-types (9) Tagged with Centronuclear myopathy (2)

Trials to join now! 8 Not yet finished but already full! 1

Gene therapy trial for rare muscle disease halted after severe side effects

Disease control Ongoing

This trial tested a gene therapy called AT132 for X-linked myotubular myopathy (XLMTM), a rare muscle disease present at birth that causes severe breathing problems. The therapy aimed to deliver a healthy copy of the MTM1 gene to improve muscle function. However, the study was st…

Phase 2/3 • Sponsor: Astellas Gene Therapies • Aim: Disease control

Last updated Sep 05, 2026 00:00 UTC

Centronuclear myopathy

Where it sits in the disease tree

Gene therapy trial for rare muscle disease halted after severe side effects