Answers are grounded in this paper and cite their sources.
Everolimus therapy shows tumor regression in cardiac rhabdomyomas of patients with tuberous sclerosis complex, suggesting an effective low-dose treatment approach.
Tuberous sclerosis complex is syndrome that affects several organs. Cardiac manifestations include rhabdomyoma, which could lead to intracardiac obstruction of blood flow. In the present case, the so far lowest documented Everolimus blood level of 2–3 ng/ml led to tumour regression. Repeated Everolimus stopping and restarting for clinical reasons serves as a proof-of-concept for Everolimus therapy in tuberous sclerosis complex.
No takes yet. Share an insight, caveat, or question.
Rupp et al. (2025) studied this question.
Synapse has enriched 10 related papers. Consider them for comparative context: