Deoxynucleoside Therapy for Thymidine Kinase 2-Deficient Myopathy.

Cristina Domínguez-GonzálezMarcos Madruga-GarridoFabiola MavillardCaterina GaroneFrancisco Javier Aguirre-RodríguezM Alice DonatiKarin KleinsteuberItxaso MartíElena Martín-HernándezJuan P Morealejo-AycinenaFrancina MunellAndrés NascimentoSusana G KalkoM Dolores SardinaConcepcion Álvarez Del VayoOlga SerranoYuelin LongYuqi TuBruce LevinJohn L P ThompsonKristen EngelstadJasim UddinJavier Torres-TorronterasCecilia Jimenez-MallebreraRamon MartíCarmen ParadasMichio Hirano

Published in: Annals of neurology (2019)

This open-label study indicates favorable side effect profiles and clinical efficacy of deoxynucleoside monophosphate and deoxynucleoside therapies for TK2 deficiency. ANN NEUROL 2019;86:293-303.

Keyphrases

open label
clinical trial
late onset
study protocol
tyrosine kinase
squamous cell carcinoma
radiation therapy
phase iii