JS Barrett, T Nicholas, K Azer, BW Corrigan - Pharmaceutical Research, 2022 - Springer
The use of Disease progression models (DPMs) in Drug Development has been widely adopted across therapeutic areas as a method for integrating previously obtained disease …
Background Progressive supranuclear palsy (PSP) is a neurodegenerative, late‐onset disease that is challenging in terms of assessment. The Progressive Supranuclear Palsy …
A Hamdan, N Hendrickx, AC Hooker… - Clinical …, 2024 - Wiley Online Library
Degenerative cerebellar ataxias comprise a heterogeneous group of rare and ultra‐rare genetic diseases. While disease‐modifying treatments are now on the horizon for many …
Background Longitudinal item response theory (IRT) models previously suggested that the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS‐UPDRS) motor …
During drug development, a key step is the identification of relevant covariates predicting between‐subject variations in drug response. The full random effects model (FREM) is one …
In the current work, we present the methodology for development of an Item Response Theory model within a non-linear mixed effects framework to characterize the longitudinal …
Pharmacokinetic-based prophylaxis of replacement factor VIII (FVIII) products has been encouraged in recent years, but the relationship between exposure (factor VIII activity) and …
NB Lincoln, LE Bradshaw… - Health Technology …, 2020 - ncbi.nlm.nih.gov
BACKGROUND People with multiple sclerosis have problems with memory and attention. The effectiveness of cognitive rehabilitation has not been established. OBJECTIVES The …
Abstract The Movement Disorder Society revised version of the Unified Parkinson's Disease Rating Scale (MDS‐UPDRS) parts 2 and 3 reflect patient‐reported functional impact and …