Open access
Jul 2026
A prospective natural history study protocol for clinical trial readiness in synaptic disorders.
A feasible natural history protocol with prospective data for two complex neurodevelopmental disorders with natural histories that have previously been incompletely characterized is presented, within a regulatory framework that will support the use of these data to expedite clinical trial development.
J. McKee, S. Ruggiero, K. Cunningham et al.
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