Experimental gene therapy aims to help boys with rare muscle disease breathe easier
NCT ID NCT07052929
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a new gene therapy called ASP2957 for X-linked myotubular myopathy (XLMTM), a rare muscle disease present at birth that often requires a ventilator to breathe. The therapy delivers a healthy copy of the MTM1 gene using a modified virus. Nine boys up to 3 years old will receive a single infusion, and researchers will monitor safety and find the best dose.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ASP2957 (a gene therapy using a modified virus to deliver a healthy MTM1 gene)
What this could lead to
If it works, this could point toward a treatment that improves muscle function and reduces the need for a ventilator in boys with XLMTM.
What could go wrong
This is the first time ASP2957 is tested in humans, so safety and effectiveness are unknown. The trial is very small (9 boys), and results may not apply to all patients. There are risks from the gene therapy and immune-suppressing drugs.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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Lurie Children's Hospital
RECRUITINGChicago, Illinois, 60611, United States
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Oregon Health & Science University
RECRUITINGPortland, Oregon, 97239, United States
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The Hospital for Sick Children
RECRUITINGToronto, Ontario, Canada