Overview

Treatment of a Single Patient With CRD-TMH-001

Status:
Enrolling by invitation
Trial end date:
2023-09-01
Target enrollment:
0
Participant gender:
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 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Cure Rare Disease, Inc
Collaborator:
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.