Amyotrophic lateral sclerosis clinical trials and interpretation of functional end points and fluid biomarkers: a review

JM Shefner, R Bedlack, JA Andrews, JD Berry… - JAMA …, 2022 - jamanetwork.com
Importance Clinical trial activity in amyotrophic lateral sclerosis (ALS) is dramatically
increasing; as a result, trial modifications have been introduced to improve efficiency …

Current state and future directions in the diagnosis of amyotrophic lateral sclerosis

M Vidovic, LH Müschen, S Brakemeier, G Machetanz… - Cells, 2023 - mdpi.com
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by
loss of upper and lower motor neurons, resulting in progressive weakness of all voluntary …

Role of the repeat expansion size in predicting age of onset and severity in RFC1 disease

R Currò, N Dominik, S Facchini, E Vegezzi, R Sullivan… - Brain, 2024 - academic.oup.com
RFC1 disease, caused by biallelic repeat expansion in RFC1, is clinically heterogeneous in
terms of age of onset, disease progression and phenotype. We investigated the role of the …

Erythrocytes' surface properties and stiffness predict survival and functional decline in ALS patients

CS Lopes, AC Pronto‐Laborinho, VA Conceição… - …, 2024 - Wiley Online Library
Erythrocytes play a fundamental role in oxygen delivery to tissues and binding to
inflammatory mediators. Evidences suggest that dysregulated erythrocyte function could …

Review of disease-modifying drug trials in amyotrophic lateral sclerosis

P Tornese, S Lalli, A Cocco, A Albanese - Journal of Neurology …, 2022 - jnnp.bmj.com
We analysed clinical trials of pharmacological interventions on patients with amyotrophic
lateral sclerosis (ALS), and compared study quality and design features. The systematic …

Dietary Intake of micronutrients and disease severity in patients with amyotrophic lateral sclerosis

ANAB Barros, MLN Felipe, IR Barbosa, L Leite-Lais… - Metabolites, 2023 - mdpi.com
Vitamins and essential metals have been studied as potential risk and prognostic factors in
amyotrophic lateral sclerosis (ALS). This study aimed to evaluate the prevalence of …

Long-term survival benefits of intrathecal autologous bone marrow-derived mesenchymal stem cells (Neuronata-R®: lenzumestrocel) treatment in ALS: Propensity …

JY Nam, S Chun, TY Lee, Y Seo, K Kim… - Frontiers in Aging …, 2023 - frontiersin.org
Objective Neuronata-R®(lenzumestrocel) is an autologous bone marrow-derived
mesenchymal stem cell (BM-MSC) product, which was conditionally approved by the Korean …

Dynamic Bayesian networks for stratification of disease progression in amyotrophic lateral sclerosis

M Gromicho, T Leão, M Oliveira Santos… - European Journal of …, 2022 - Wiley Online Library
Background and purpose Progression rate is quite variable in amyotrophic lateral sclerosis
(ALS); thus, tools for profiling disease progression are essential for timely interventions. The …

Clinical and neurophysiological biomarkers of disease progression in amyotrophic lateral sclerosis

A Hannaford, K Byth, N Pavey, RD Henderson… - Muscle & …, 2023 - Wiley Online Library
Abstract Introduction/Aims Rate of disease progression (ΔFS), measured as change in the
revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS‐R) and body mass …

Functional lingual pressure thresholds for swallowing safety and efficiency impairments in amyotrophic lateral sclerosis

R Robison, L DiBiase, JP Wymer, EK Plowman - Dysphagia, 2023 - Springer
Although reductions in lingual strength are reported in individuals with amyotrophic lateral
sclerosis (ALS) that are associated with dysphagia; determination of a functional lingual …