A Long-term Follow-up Study of an Open-label, Single-arm, Single-dose Escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intracerebroventricular Injection of RB001 in Participants With SHANK3-related Phelan-McDermid Syndrome.

Publication date: Jul 01, 2026

This is a long-term follow-up study of participants in the RB001-101 gene replacement therapy clinical trial for Children With SHANK3-related Phelan-McDermid Syndrome (PMS). Participants will roll over from the parent study into this long-term study for continuous safety monitoring and preliminary efficacy assessment for up to 5 years.

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Original Article