The clinical pharmacology of cladribine tablets for the treatment of relapsing multiple sclerosis

R Hermann, MO Karlsson, AM Novakovic… - Clinical …, 2019 - Springer
Abstract Cladribine Tablets (MAVENCLAD®) are used to treat relapsing multiple sclerosis
(MS). The recommended dose is 3.5 mg/kg, consisting of 2 annual courses, each comprising …

Role of disease progression models in drug development

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 …

Quantitative comparisons of progressive supranuclear palsy rating scale versions using item response theory

M Gewily, EL Plan, E Yousefi, F König… - Movement …, 2024 - Wiley Online Library
Background Progressive supranuclear palsy (PSP) is a neurodegenerative, late‐onset
disease that is challenging in terms of assessment. The Progressive Supranuclear Palsy …

Longitudinal analysis of natural history progression of rare and ultra‐rare cerebellar ataxias using item response theory

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 …

Dissecting the domains of Parkinson's disease: insights from longitudinal item response theory modeling

S Luo, H Zou, GT Stebbins… - Movement …, 2022 - Wiley Online Library
Background Longitudinal item response theory (IRT) models previously suggested that the
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS‐UPDRS) motor …

Properties of the full random‐effect modeling approach with missing covariate data

J Nyberg, EN Jonsson, MO Karlsson… - Statistics in …, 2024 - Wiley Online Library
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 …

Modeling a composite score in Parkinson's disease using item response theory

G Gottipati, MO Karlsson, EL Plan - The AAPS journal, 2017 - Springer
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 …

Relationship between factor VIII activity, bleeds and individual characteristics in severe hemophilia A patients

JA Abrantes, A Solms, D Garmann, EI Nielsen… - …, 2019 - pmc.ncbi.nlm.nih.gov
Pharmacokinetic-based prophylaxis of replacement factor VIII (FVIII) products has been
encouraged in recent years, but the relationship between exposure (factor VIII activity) and …

[HTML][HTML] Group cognitive rehabilitation to reduce the psychological impact of multiple sclerosis on quality of life: the CRAMMS RCT.

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 …

Application of longitudinal item response theory models to modeling Parkinson's disease progression

H Zou, V Aggarwal, GT Stebbins… - CPT …, 2022 - Wiley Online Library
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 …