Long-term safety of dexamethasone sodium phosphate encapsulated in autologous erythrocytes in pediatric patients with ataxia telangiectasia.

Journal: Frontiers in neurology

Publication Year: 

Authors & Co-authors:  Koenig Mary Kay MK Leuzzi Vincenzo V Gouider Riadh R Yiu Eppie M EM Pietrucha Barbara B Stray-Pedersen Asbjørg A Perlman Susan L SL Wu Steve S Burgers Trudy T Borgohain Rupam R

Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia (ATTeST): a multicentre, randomised, double-blind, placebo-controlled phase 3 trial.

Journal: The Lancet. Neurology

Publication Year: 2024

Authors & Co-authors:  Zielen Stefan S Crawford Thomas T Benatti Luca L Magnani Mauro M Kieslich Matthias M Ryan Monique M Meyts Isabelle I Gulati Sheffali S Borgohain Rupam R Yadav Ravi R