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SHANK3 Haploinsufficiency
Clinical trial pipeline · Data from ClinicalTrials.gov
See which SHANK3 Haploinsufficiency trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which SHANK3 Haploinsufficiency trials may be worth asking aboutNorth America
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This is an open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of RB001 administered via intracerebro…
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 Sy…