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Completed
NCT05514249
Treatment of a Single Patient With CRD-TMH-001
Conditions: Duchenne Muscular Dystrophy
Sex: Male
Ages: 18 Years – 28 Years
Healthy volunteers: No
Phase: PHASE1
Enrollment: 1
Sponsor: Cure Rare Disease, Inc
Location: UMass Chan Medical School Worcester Massachusetts
Summary
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.
Eligibility Criteria
Inclusion Criteria:
* Completion of informed consent
* Confirmation of genetic mutation
* Confirmation of absence of elevated AAV9 NAbs
Exclusion Criteria:
\- Any significant medical issue(s) (past or current) that would, in the opinion of the Principal Investigator (PI), prevent this patient from being dosed.
Source: ClinicalTrials.gov (NCT05514249). StuddyBuddy aggregates publicly available trial information.
