Last Updated: 09/26/2026
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Found 52 publications
Speech-based digital endpoints track ALS progression and align with standard clinical outcomes: evidence from the VRG50635 trial.
Journal: Scientific reports
Published: February 04, 2026
Glycoprotein Non-Metastatic Melanoma Protein B (GPNMB): A Translational Pharmacodynamic Biomarker for PIKfyve Inhibition With VRG50635.
Journal: Clinical and translational science
Published: October 07, 2025
Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial.
Journal: The Lancet. Neurology
Published: July 19, 2023
The feasibility and utility of hair follicle sampling to measure FMRP and FMR1 mRNA in children with or without fragile X syndrome: a pilot study.
Journal: Journal of neurodevelopmental disorders
Published: November 30, 2021
Quantitative muscle analysis in facioscapulohumeral muscular dystrophy using whole-body fat-referenced MRI: Protocol development, multicenter feasibility, and repeatability.
Journal: Muscle & nerve
Published: September 23, 2021
Quantitative Muscle Analysis in FSHD Using Whole-Body Fat-Referenced MRI: Composite Scores for Longitudinal and Cross-sectional Analysis.
Journal: Neurology
Published: September 13, 2021
FMRP Levels in Human Peripheral Blood Leukocytes Correlates with Intellectual Disability.
Journal: Diagnostics (Basel, Switzerland)
Published: July 18, 2021
Social communication in fragile X syndrome: pilot examination of the Brief Observation of Social Communication Change (BOSCC).
Journal: Journal of neurodevelopmental disorders
Published: April 05, 2021
Phase 1 clinical trial of losmapimod in facioscapulohumeral dystrophy: Safety, tolerability, pharmacokinetics, and target engagement.
Journal: British journal of clinical pharmacology
Published: December 21, 2020
Gray Matter Nucleus Hyperintensity After Monthly Triple-Dose Gadopentetate Dimeglumine With Long-term Magnetic Resonance Imaging.
Journal: Investigative radiology
Published: September 08, 2020
p38α Regulates Expression of DUX4 in a Model of Facioscapulohumeral Muscular Dystrophy.
Journal: The Journal of pharmacology and experimental therapeutics
Published: December 16, 2019
Evoked potentials as a translatable biomarker to track functional remyelination.
Journal: Molecular and cellular neurosciences
Published: April 22, 2019
Last Updated: 09/26/2026