Clinical and histologic analysis of the efficacy of topical rapamycin therapy against hypomelanotic macules in tuberous sclerosis complex
M Wataya-Kaneda, M Tanaka, L Yang, F Yang… - JAMA …, 2015 - jamanetwork.com
Importance Tuberous sclerosis complex (TSC) is an autosomal dominant disorder leading to
the aberrant activation of the mammalian target of rapamycin complex 1. Although the
efficacy of mammalian target of rapamycin complex 1 inhibitors against tumors in patients
with TSC, including facial angiofibroma, has been well investigated, their efficacy against
hypomelanotic macules in patients with TSC is unknown. Objectives To evaluate objectively
the efficacy of topical rapamycin treatment of hypomelanotic macules in patients with TSC …
the aberrant activation of the mammalian target of rapamycin complex 1. Although the
efficacy of mammalian target of rapamycin complex 1 inhibitors against tumors in patients
with TSC, including facial angiofibroma, has been well investigated, their efficacy against
hypomelanotic macules in patients with TSC is unknown. Objectives To evaluate objectively
the efficacy of topical rapamycin treatment of hypomelanotic macules in patients with TSC …