[HTML][HTML] Electrophysiological and Imaging Biomarkers to Evaluate Exercise Training in Patients with Neuromuscular Disease: A Systematic Review

L Pomp, JAL Jeneson, WL van der Pol… - Journal of Clinical …, 2023 - mdpi.com
Exercise therapy as part of the clinical management of patients with neuromuscular
diseases (NMDs) is complicated by the limited insights into its efficacy. There is an urgent …

Quantitative muscle ultrasound in adult spinal muscular atrophy. A pilot study

L Pelosi, M Rodrigues, C Zhong, S Patel… - Muscle & …, 2024 - Wiley Online Library
Abstract Introduction/Aims Muscle ultrasound has been investigated in children with spinal
muscular atrophy (SMA) and proposed as a potential biomarker of disease severity. We …

Preemptive dual therapy for children at risk for infantile‐onset spinal muscular atrophy

SE Matesanz, KW Brigatti, M Young… - Annals of Clinical …, 2024 - Wiley Online Library
Objective Compare efficacy of gene therapy alone (monotherapy) or in combination with an
SMN2 augmentation agent (dual therapy) for treatment of children at risk for spinal muscular …

Development of an ultrasound‐based metric of muscle functional capacity for use in patients with neuromuscular disease

AN McCrady, CD Masterson, LE Barnes, RJ Scharf… - Muscle & … - Wiley Online Library
Abstract Introduction/Aims Spinal muscular atrophy (SMA) and Duchenne muscular
dystrophy (DMD) are progressive neuromuscular disorders characterized by severe muscle …