First human test of gene therapy for rare muscle disease begins
NCT ID NCT05230459
Summary
This study is testing the safety of a single-dose gene therapy called AB-1003 in adults with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle disorder. Researchers will give increasing doses to small groups of participants to check for side effects. The goal is to see if this experimental treatment is safe enough for further testing.
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Contacts and locations
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Study contacts
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Locations
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Kennedy Krieger Institute
RECRUITINGBaltimore, Maryland, 21205, United States
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University of California - Irvine
RECRUITINGIrvine, California, 92697, United States
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
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University of Kansas Medical Center
RECRUITINGKansas City, Kansas, 66160, United States
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University of Washington Medical Center
RECRUITINGSeattle, Washington, 98195, United States
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VCU
RECRUITINGRichmond, Virginia, 23298, United States
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Conditions
Explore the condition pages connected to this study.