Preventative treatment of tuberous sclerosis complex with sirolimus: Phase I safety and efficacy results

• Main Authors: Jamie K. Capal (Author), David M. Ritter (Author), David Neal Franz (Author), Molly Griffith (Author), Kristn Currans (Author), Bridget Kent (Author), E. Martina Bebin (Author), Hope Northrup (Author), Mary Kay Koenig (Author), Tomoyuki Mizuno (Author), Alexander A. Vinks (Author), Stephanie L. Galandi (Author), Wujuan Zhang (Author), Kenneth D.R. Setchell (Author), Kelly M. Kremer (Author), Carlos M. Prada (Author), Hansel M. Greiner (Author), Katherine Holland‐Bouley (Author), Paul S. Horn (Author), Darcy A. Krueger (Author)
• Format: Book
• Published: Wiley, 2024-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Objective Tuberous sclerosis complex (TSC) results from overactivity of the mechanistic target of rapamycin (mTOR). Sirolimus and everolimus are mTOR inhibitors that treat most facets of TSC but are understudied in infants. We sought to understand the safety and potential efficacy of preven...