Are we prepared for clinical trials in Charcot-Marie-Tooth disease?-Reference-Cited by-同舟云学术

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Are we prepared for clinical trials in Charcot-Marie-Tooth disease?

Published:2020-02 Issue: Volume:1729 Page:146625
ISSN:0006-8993
Container-title:Brain Research
language:en
Short-container-title:Brain Research

Author:

Rossor A.M.,Shy M.E.,Reilly M.M.

Funder

Medical Research Council

MRC Centre

National Institutes of Neurological Diseases

Stroke and office of Rare Diseases

National Institute for Health Research University College London Hospitals Biomedical Research Centre

Publisher

Elsevier BV

Subject

Developmental Biology,Neurology (clinical),Molecular Biology,General Neuroscience

Reference48 articles.

1. An exploratory randomised double-blind and placebo-controlled phase 2 study of a combination of baclofen, naltrexone and sorbitol (PXT3003) in patients with Charcot-Marie-Tooth disease type 1A;Attarian;Orphanet J. Rare Dis.,2014

2. Motor unit number index correlates with disability in Charcot-Marie-Tooth disease;Bas;Clin. Neurophysiol.,2018

3. Serum neurofilament light chain: a biomarker of neuronal injury in vasculitic neuropathy;Bischof;Ann. Rheum. Dis.,2018

4. Validation of the Charcot-Marie-Tooth disease pediatric scale as an outcome measure of disability;Burns;Ann. Neurol.,2012

5. Ascorbic acid for Charcot-Marie-Tooth disease type 1A in children: a randomised, double-blind, placebo-controlled, safety and efficacy trial;Burns;Lancet. Neurol.,2009

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1. p62/sequestosome-1 as a severity-reflecting plasma biomarker in Charcot–Marie–Tooth disease type 1A;Scientific Reports;2024-05-14

2. One-Year Longitudinal Assessment of Patients With CMT1A Using Quantitative MRI;Neurology;2024-05

3. Charcot-Marie-tooth disease type 2A: An update on pathogenesis and therapeutic perspectives;Neurobiology of Disease;2024-04

4. Parent‐proxy pediatric CMT quality of life outcome measure: Validation of the Italian version;Journal of the Peripheral Nervous System;2024-02-08

5. Quantitative MRI outcome measures in CMT1A using automated lower limb muscle segmentation;Journal of Neurology, Neurosurgery & Psychiatry;2023-11-18

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