Rational Design of Novel Therapies for Pantothenate Kinase-Associated Neurodegeneration.

Nivedita ThakurThomas KlopstockSuzanne JackowskiEnej KuscerFernando TrictaAleksandar VidenovicHyder A Jinnah

Published in: Movement disorders : official journal of the Movement Disorder Society (2021)

Experience with these trials has encouraged a critical evaluation of optimal trial designs, as well as the development of PKAN-specific measures to monitor outcomes. PKAN provides a valuable example for understanding targeted drug development and clinical trial design for rare disorders. © 2021 International Parkinson and Movement Disorder Society.

Keyphrases

clinical trial
phase ii
phase iii
study protocol
open label
cancer therapy
double blind
protein kinase
randomized controlled trial
tyrosine kinase
drug delivery
skeletal muscle
adipose tissue
finite element analysis
glycemic control
insulin resistance