The era of cryptic exons: implications for ALS-FTD

PR Mehta, AL Brown, ME Ward, P Fratta - Molecular Neurodegeneration, 2023 - Springer
TDP-43 is an RNA-binding protein with a crucial nuclear role in splicing, and mislocalises
from the nucleus to the cytoplasm in a range of neurodegenerative disorders. TDP-43 …

Current state and future directions in the therapy of ALS

L Tzeplaeff, S Wilfling, MV Requardt, M Herdick - Cells, 2023 - mdpi.com
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder
affecting upper and lower motor neurons, with death resulting mainly from respiratory failure …

Blood-based biomarkers of inflammation in amyotrophic lateral sclerosis

KA Staats, DR Borchelt, MG Tansey… - Molecular …, 2022 - Springer
Abstract Amyotrophic Lateral Sclerosis (ALS) is a devastating neurodegenerative disease in
which many processes are detected including (neuro) inflammation. Many drugs have been …

Blood biomarkers in ALS: challenges, applications and novel frontiers

E Sturmey, A Malaspina - Acta Neurologica Scandinavica, 2022 - Wiley Online Library
Amyotrophic lateral sclerosis (ALS) is the most common motor neuron disease among
adults. With diagnosis reached relatively late into the disease process, extensive motor cell …

[PDF][PDF] Disease state overview

D Hulisz - Am J Manag Care, 2018 - ajmc.s3.amazonaws.com
or swallowing. 1 Signs of bulbar disease include dysarthria, facial weakness, weakness
pushing the tongue outward (pulsion), poor palate elevation, difficulty chewing, impaired …

Neurofilaments in motor neuron disorders: towards promising diagnostic and prognostic biomarkers

E Zucchi, V Bonetto, G Sorarù, I Martinelli… - Molecular …, 2020 - Springer
Motor neuron diseases (MNDs) are etiologically and biologically heterogeneous diseases.
The pathobiology of motor neuron degeneration is still largely unknown, and no effective …

Biomarkers in motor neuron disease: a state of the art review

NS Verber, SR Shepheard, M Sassani… - Frontiers in …, 2019 - frontiersin.org
Motor neuron disease can be viewed as an umbrella term describing a heterogeneous
group of conditions, all of which are relentlessly progressive and ultimately fatal. The …

Predictors of survival in patients with amyotrophic lateral sclerosis: a large meta-analysis

WM Su, YF Cheng, Z Jiang, QQ Duan, TM Yang… - …, 2021 - thelancet.com
Background The survival time of amyotrophic lateral sclerosis (ALS) is greatly variable and
protective or risk effects of the potential survival predictors are controversial. Thus, we aim to …

Efficacy and safety of ultrahigh-dose methylcobalamin in early-stage amyotrophic lateral sclerosis: a randomized clinical trial

R Oki, Y Izumi, K Fujita, R Miyamoto, H Nodera… - JAMA …, 2022 - jamanetwork.com
Importance The effectiveness of currently approved drugs for amyotrophic lateral sclerosis
(ALS) is restricted; there is a need to develop further treatments. Initial studies have shown …

Addressing heterogeneity in amyotrophic lateral sclerosis CLINICAL TRIALS

NA Goyal, JD Berry, A Windebank, NP Staff… - Muscle & …, 2020 - Wiley Online Library
Amyotrophic lateral sclerosis (ALS) is a debilitating neurodegenerative disorder with
complex biology and significant clinical heterogeneity. Many preclinical and early phase …