Rapamycin and rapalogs for tuberous sclerosis complex

TH Sasongko, K Kademane, SCS Hou… - Cochrane Database …, 2023 - cochranelibrary.com
Background Potential benefits of rapamycin or rapalogs for treating people with tuberous
sclerosis complex (TSC) have been shown. Currently everolimus (a rapalog) is only …

Topical use of mammalian target of rapamycin inhibitors in dermatology: a systematic review with meta-analysis

S Leducq, B Giraudeau, E Tavernier… - Journal of the American …, 2019 - Elsevier
Background Systemic mammalian target of rapamycin (mTOR) inhibitors are currently used
in many dermatologic indications. Their topical use is recent and poorly codified. Objective …

[HTML][HTML] Topical rapamycin therapy to alleviate the cutaneous manifestations of tuberous sclerosis complex: a double-blind, randomized, controlled trial to evaluate the …

MK Koenig, AA Hebert, J Roberson, J Samuels… - Drugs in R&d, 2012 - Springer
Abstract Background and Objectives Facial angiofibromas are disfiguring facial lesions,
present in up to 80% of patients with tuberous sclerosis complex. Recent elucidation of the …

Efficacy and safety of topical sirolimus therapy for facial angiofibromas in the tuberous sclerosis complex: a randomized clinical trial

M Wataya-Kaneda, A Nakamura, M Tanaka… - JAMA …, 2017 - jamanetwork.com
Importance Inhibitors of mammalian target of rapamycin complex 1, such as sirolimus,
effectively target skin lesions in tuberous sclerosis complex (TSC). However, systemic …

A novel rapamycin cream formulation improves facial angiofibromas associated with tuberous sclerosis complex: a double-blind randomized placebo-controlled trial

P Aitken, I Stanescu, L Boddington… - British Journal of …, 2023 - academic.oup.com
Abstract Background Facial angiofibromas (FAs) are a major feature of tuberous sclerosis
complex (TSC). Topical rapamycin can successfully treat FAs. A new stabilized cream …

Sirolimus gel treatment vs placebo for facial angiofibromas in patients with tuberous sclerosis complex: a randomized clinical trial

M Wataya-Kaneda, Y Ohno, Y Fujita… - JAMA …, 2018 - jamanetwork.com
Importance Most patients with tuberous sclerosis complex (TSC), an autosomal-dominant
disorder that is caused by the constitutive activation of mammalian target of rapamycin …

Analysis of current data on the use of topical rapamycin in the treatment of facial angiofibromas in tuberous sclerosis complex

R Balestri, I Neri, A Patrizi, L Angileri… - Journal of the …, 2015 - Wiley Online Library
Background Tuberous sclerosis complex (TSC) is an autosomal dominant neurocutaneous
syndrome causing hamartomatous growths in multiple organs. Facial angiofibromas occur in …

First left–right comparative study of topical rapamycin vs. vehicle for facial angiofibromas in patients with tuberous sclerosis complex

M Tanaka, M Wataya‐Kaneda… - British Journal of …, 2013 - academic.oup.com
Background Tuberous sclerosis complex (TSC) is an autosomal dominant disorder causing
multiple hamartomas. Treatment of TSC lesions with mammalian target of rapamycin …

Long-term treatment of cutaneous manifestations of tuberous sclerosis complex with topical 1% sirolimus cream: a prospective study of 25 patients

N Malissen, L Vergely, M Simon, A Roubertie… - Journal of the American …, 2017 - Elsevier
Background Data on long-term topical sirolimus treatment of the cutaneous manifestations of
tuberous sclerosis complex are rare. Objective To evaluate the long-term benefit and …

Tuberous sclerosis complex: an update for dermatologists

SK Jacks, PM Witman - Pediatric Dermatology, 2015 - Wiley Online Library
Tuberous sclerosis complex is an autosomal dominant disorder that often manifests early in
life with cutaneous features, and it is important that dermatologists who care for children …