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About
The study is a single patient study intended to understand the effects of a gene-editing therapeutic to treat a rare mutation of Duchenne muscular dystrophy.
Full description
The objective of the study is to assess the safety and preliminary efficacy of CRD-TMH-001 after intravenous administration for a period of 1 year with long-term follow-up out to 15 years.
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Interventional model
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1 participants in 1 patient group
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Data sourced from clinicaltrials.gov
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