Charlotte Lilien
Oxford, ENG, United Kingdom
Last Updated: 09/26/2026
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Found 15 publications
Rehabilitation research in spinal muscular atrophy: a call to action.
Journal: Journal of neuromuscular diseases
Published: August 25, 2025
A systematic review on motor outcome measures in congenital myopathy.
Journal: Neuromuscular disorders : NMD
Published: February 26, 2025
Pushing the boundaries: future directions in the management of spinal muscular atrophy.
Journal: Trends in molecular medicine
Published: October 07, 2024
Patients' Perceptions of Nusinersen Effects According to Their Responder Status.
Journal: Journal of clinical medicine
Published: May 09, 2024
An algorithm for discontinuing mechanical ventilation in boys with x-linked myotubular myopathy after positive response to gene therapy: the ASPIRO experience.
Journal: Respiratory research
Published: February 13, 2024
Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative.
Journal: Journal of neuromuscular diseases
Published: October 31, 2022
INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.
Journal: Journal of neuromuscular diseases
Published: June 13, 2022
Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy.
Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy.
Journal: Annals of clinical and translational neurology
Published: April 01, 2021
Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study.
Journal: Annals of clinical and translational neurology
Published: October 15, 2020
Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance.
Journal: Journal of neuromuscular diseases
Published: September 14, 2020
Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy.
Journal: Orphanet journal of rare diseases
Published: June 23, 2020
French adaptation of motor function scales for patients with spinal muscular atrophy
Journal: Medecine sciences : M/S
Published: December 21, 2019
Last Updated: 09/26/2026