RecruitingNot applicableNCT06615206

A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)

Studying Proximal Xq28 duplication syndrome

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Key facts

Sponsor
HuidaGene Therapeutics Co., Ltd.
Principal Investigator
Study Director
Intervention
HG204(genetic)
Enrollment
6 enrolled
Eligibility
2-18 years · MALE
Timeline
20242026

Study locations (1)

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

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