Long-term follow-up for RB001 gene therapy in children with Phelan-McDermid syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study follows children who completed the RB001 gene therapy trial for Phelan-McDermid syndrome (caused by changes in the SHANK3 gene) to monitor their health and progress over a longer period of time.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have Phelan-McDermid syndrome caused by a change in the SHANK3 gene.
- Your child must have completed the RB001-101 study, including the final check-up at week 52 after receiving the gene therapy.
- There are no other specific reasons that the study doctor thinks would make participation unsafe or unsuitable for your child.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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