Clinical Trial Readiness in Limb Girdle Muscular Dystrophy R1 (LGMDR1): A GRASP Consortium Study
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Identifying functional measures that are both valid and reliable in the limb girdle muscular dystrophy (LGMD) population is critical for quantifying the level of functional impairment related to disease progression in order to establish clinical trial readiness in the context of anticipated therapeutic trials.
Stephanie M. Hunn +29 more
wiley +1 more source
Estimating Treatment Effect Heterogeneity in Psychiatry: A Review and Tutorial With Causal Forests. [PDF]
Int J Methods Psychiatr ResSverdrup E, Petukhova M, Wager S.
europepmc +1 more source
Some renewal theorems for non-negative independent random variables [PDF]
, 1965 John Williamson
openalex +1 more source
ADAPT NXT: Fixed Cycles or Every‐Other‐Week IV Efgartigimod in Generalized Myasthenia Gravis
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective This phase 3b, open‐label, randomized ADAPT NXT study investigated the efficacy, safety, and tolerability of efgartigimod administered in either a fixed cycles dosing regimen (3 cycles of 4 once‐weekly infusions, with 4 weeks between cycles) or a cycle followed by every‐other‐week (Q2W) dosing.
Ali A. Habib +16 more
wiley +1 more source
Susceptibility to Photosynthesis Suppression From Extreme Storms Is Highly Site-Dependent. [PDF]
Glob Chang BiolMcCormick EL +4 more
europepmc +1 more source
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT This study assessed whether continued treatment with anti‐CGRP monoclonal antibodies (mAbs) is driven more by reductions in monthly migraine days (MMDs) or patients' global impression of change (PGIC), a patient‐reported outcome. Among 169 patients treated with anti‐CGRP mAbs, 21.3% discontinued due to ineffectiveness. PGIC responders (≥ 5) at
Marina Romozzi +4 more
wiley +1 more source
Predictive factors of adolescents' happiness: a random forest analysis of the 2023 Korea Youth Risk Behavior Survey. [PDF]
Child Health Nurs ResKim EJ, Kim SK, Jung SH, Ryu YS.
europepmc +1 more source
Validity and Reliability of Clinical and Patient‐Reported Outcomes in Multisystem Proteinopathy 1
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Valosin‐containing protein (VCP)‐associated multisystem proteinopathy 1 (MSP1) is caused by variants in the VCP gene. MSP1 results in various phenotypes including progressive myopathy, Paget's disease of bone, frontotemporal dementia, amyotrophic lateral sclerosis, and parkinsonism, among others.
Lindsay N. Alfano +15 more
wiley +1 more source
Analysis of influencing factors and equity in health education for mobile populations based on random forest model: evidence from the China migrants dynamic survey. [PDF]
Front Public HealthJiang MH, Lei SY, Yang F.
europepmc +1 more source