Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial
Under a Creative Commons license
open access
Abbreviations
ACMG
American College of Medical Genetics and Genomics
ADHD RS-IV IA
Attention Deficit Hyperactivity Disorder Rating Scale IV, inattention subscore
AE
adverse event
ANCOVA
analysis of covariance
CANTAB
Cambridge Neuropsychological Test Automated Battery
CI
confidence interval
CTCAE
Common Terminology Criteria for Adverse Events
HAE
hypersensitivity adverse events
LS
least squares
MedDRA
Medical Dictionary for Regulatory Activities
mITT
modified intent-to-treat
MMRM
mixed model repeated measures
NIAID/FAAN
National Institute of Allergy and Infectious Diseases/Food Allergy and Anaphylaxis Network
PAH
phenylalanine hydroxylase
PAL
phenylalanine ammonia lyase
PEG
polyethylene glycol
Phe
phenylalanine
PKU
phenylketonuria
POMS
Profile of Mood States
RDT
randomized discontinuation trial
RVP
rapid visual processing
SD
standard deviation
SMQ
standardized MedDRA query
SST
stop signal task
SWM
spatial working memory
Keywords
Phenylketonuria
PKU
Recombinant Anabaena variabilis PEGylated phenylalanine ammonia lyase
Pegvaliase
Cited by (0)
© 2018 The Authors. Published by Elsevier Inc.