ReviewJournal of neuromuscular diseases2026
X-linked myotubular myopathy, liver disease, and gene therapy.
Review in Journal of neuromuscular diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
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0 citing papers in PubMed.
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Authors and funding
4 authors.
Funding
Abstract
X-linked myotubular myopathy (XLMTM) is a rare and severe neuromuscular disorder commonly characterized by profound skeletal muscle weakness, significant co-morbid disabilities, and early death. XLMTM has traditionally been viewed as primarily a disorder of skeletal muscle. However, there is an emerging appreciation of extra-muscular symptom involvement in XLMTM, most notably hepatobiliary disease. As promising genetic therapies for XLMTM rapidly advance, incomplete understanding of these manifestations has presented a significant barrier to safe and effective clinical translation, as well limited understanding of disease management and patient outcomes. In this review, we provide an update on the recent advances in XLMTM drug development, examine emerging data on XLMTM-associated liver disease and its impact on therapeutic development, and discuss future directions for understanding and targeting the serious non-muscle manifestations of this disorder.
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