Amyotrophic lateral sclerosis

EL Feldman, SA Goutman, S Petri, L Mazzini… - The Lancet, 2022 - thelancet.com
Amyotrophic lateral sclerosis is a fatal CNS neurodegenerative disease. Despite intensive
research, current management of amyotrophic lateral sclerosis remains suboptimal from …

[HTML][HTML] Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation

RJ Mead, N Shan, HJ Reiser, F Marshall… - Nature Reviews Drug …, 2023 - nature.com
Amyotrophic lateral sclerosis (ALS) is a devastating disease caused by degeneration of
motor neurons. As with all major neurodegenerative disorders, development of disease …

[HTML][HTML] Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

TM Miller, ME Cudkowicz, A Genge… - … England Journal of …, 2022 - Mass Medical Soc
Background The intrathecally administered antisense oligonucleotide tofersen reduces
synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with …

The Alzheimer's Association appropriate use recommendations for blood biomarkers in Alzheimer's disease

O Hansson, RM Edelmayer, AL Boxer… - Alzheimer's & …, 2022 - Wiley Online Library
Blood‐based markers (BBMs) have recently shown promise to revolutionize the diagnostic
and prognostic work‐up of Alzheimer's disease (AD), as well as to improve the design of …

Blood biomarkers for Alzheimer's disease in clinical practice and trials

O Hansson, K Blennow, H Zetterberg, J Dage - Nature aging, 2023 - nature.com
Blood-based biomarkers hold great promise to revolutionize the diagnostic and prognostic
work-up of Alzheimer's disease (AD) in clinical practice. This is very timely, considering the …

[HTML][HTML] Trial of sodium phenylbutyrate–taurursodiol for amyotrophic lateral sclerosis

S Paganoni, EA Macklin, S Hendrix… - … England Journal of …, 2020 - Mass Medical Soc
Background Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal
death in experimental models. The efficacy and safety of a combination of the two …

[HTML][HTML] A fluid biomarker reveals loss of TDP-43 splicing repression in presymptomatic ALS–FTD

KE Irwin, P Jasin, KE Braunstein, IR Sinha, MA Garret… - Nature medicine, 2024 - nature.com
Although loss of TAR DNA-binding protein 43 kDa (TDP-43) splicing repression is well
documented in postmortem tissues of amyotrophic lateral sclerosis (ALS) and frontotemporal …

Blood neurofilament light: a critical review of its application to neurologic disease

C Barro, T Chitnis, HL Weiner - Annals of clinical and …, 2020 - Wiley Online Library
Neuronal injury is a universal event that occurs in disease processes that affect both the
central and peripheral nervous systems. A blood biomarker linked to neuronal injury would …

Detecting amyloid positivity in early Alzheimer's disease using combinations of plasma Aβ42/Aβ40 and p‐tau

S Janelidze, S Palmqvist, A Leuzy… - Alzheimer's & …, 2022 - Wiley Online Library
Introduction We studied usefulness of combining blood amyloid beta (Aβ) 42/Aβ40,
phosphorylated tau (p‐tau) 217, and neurofilament light (NfL) to detect abnormal brain Aβ …

Recent advances in the diagnosis and prognosis of amyotrophic lateral sclerosis

SA Goutman, O Hardiman, A Al-Chalabi… - The Lancet …, 2022 - thelancet.com
The diagnosis of amyotrophic lateral sclerosis can be challenging due to its heterogeneity in
clinical presentation and overlap with other neurological disorders. Diagnosis early in the …