First-in-Human gene therapy offers hope for babies on ventilators
NCT ID NCT07052929
Summary
This is the first time researchers are testing a new gene therapy called ASP2957 in people. It aims to treat a rare, severe muscle disease called X-linked myotubular myopathy (XLMTM) in very young boys who depend on a ventilator to breathe. The study will first find a safe dose and then see if delivering a healthy gene can improve muscle function.
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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
Conditions
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