Overview
Treatment of a Single Patient With CRD-TMH-001
Status:
Enrolling by invitation
Enrolling by invitation
Trial end date:
2023-09-01
2023-09-01
Target enrollment:
0
0
Participant gender:
Male
Male
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.Phase:
Phase 1Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
Cure Rare Disease, IncCollaborator:
University of Massachusetts, Worcester
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.