Long-Term Follow-Up Study for RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)

NCT07690527 · clinicaltrials.gov ↗

PHASE1

Phase

ENROLLING_BY_INVITATION

Status

8

Enrollment

OTHER

Sponsor class

Conditions

SHANK3 Haploinsufficiency
Phelan-McDermid Syndrome

Interventions

BIOLOGICAL: RB001

Sponsor

Peking University First Hospital

Collaborators

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