Effectiveness and safety of everolimus treatment in patients with tuberous sclerosis complex in real-world clinical practice

Effectiveness and safety of everolimus treatment in patients with tuberous sclerosis complex in real-world clinical practice

Abstract Background The randomised double-blinded placebo-controlled EXIST-1–3 studies have showed everolimus effective with adverse effects reported as acceptable in treatment of symptoms in patients with tuberous sclerosis complex (TSC), although evidence of outcomes in clinical practice remains l...

Full description

Bibliographic Details
Main Authors: Ine Cockerell, Jakob Christensen, Christina E. Hoei-Hansen, Lotte Holst, Mikkel Grenaa Frederiksen, Aart Imran Issa-Epe, Bård Nedregaard, Ragnar Solhoff, Ketil Heimdal, Cecilie Johannessen Landmark, Caroline Lund, Terje Nærland
Format: Article
Language:English
Published: BMC 2023-12-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Tuberous sclerosis complex
mTOR inhibitor
Everolimus
Sirolimus
Subependymal giant cell astrocytoma
Renal angiomyolipoma
Online Access:https://doi.org/10.1186/s13023-023-02982-1

Internet

https://doi.org/10.1186/s13023-023-02982-1

Similar Items