ArticleOrphanet journal of rare diseases2025

Long-term neuropsychologic outcome of pre-emptive mTOR inhibitor treatment in children with tuberous sclerosis complex (TSC) under 4 months of age (PROTECT), a two-arm, randomized, observer-blind, controlled phase IIb national multicentre clinical trial: study protocol.

Jan H Driedger et al.PubMed ↗Full text ↗Publisher ↗

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