[HTML][HTML] ALS genetics, mechanisms, and therapeutics: where are we now?

R Mejzini, LL Flynn, IL Pitout, S Fletcher… - Frontiers in …, 2019 - frontiersin.org
The scientific landscape surrounding amyotrophic lateral sclerosis (ALS) continues to shift
as the number of genes associated with the disease risk and pathogenesis, and the cellular …

[HTML][HTML] iPSC-based disease modeling and drug discovery in cardinal neurodegenerative disorders

H Okano, S Morimoto - Cell Stem Cell, 2022 - cell.com
It has been 15 years since the birth of human induced pluripotent stem cell (iPSC)
technology in 2007, and the scope of its application has been expanding. In addition to the …

[HTML][HTML] Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

VA Korobeynikov, AK Lyashchenko… - Nature medicine, 2022 - nature.com
Fused in sarcoma (FUS) is an RNA-binding protein that is genetically and pathologically
associated with rare and aggressive forms of amyotrophic lateral sclerosis (ALS) and …

[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 …

Riluzole and edaravone: A tale of two amyotrophic lateral sclerosis drugs

MK Jaiswal - Medicinal Research Reviews, 2019 - Wiley Online Library
Over the past decades, a multitude of experimental drugs have been shown to delay
disease progression in preclinical animal models of amyotrophic lateral sclerosis (ALS) but …

Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial

K Abe, M Aoki, S Tsuji, Y Itoyama, G Sobue… - The Lancet …, 2017 - thelancet.com
Background In a previous phase 3 study in patients with amyotrophic lateral sclerosis (ALS),
edaravone did not show a significant difference in the Revised ALS Functional Rating Scale …

SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

C Mueller, JD Berry, DM McKenna-Yasek… - … England Journal of …, 2020 - Mass Medical Soc
Two patients with familial amyotrophic lateral sclerosis (ALS) and mutations in the gene
encoding superoxide dismutase 1 (SOD1) were treated with a single intrathecal infusion of …

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 …

Transfer learning in brain-computer interfaces

V Jayaram, M Alamgir, Y Altun… - IEEE Computational …, 2016 - ieeexplore.ieee.org
The performance of brain-computer interfaces (BCIs) improves with the amount of available
training data; the statistical distribution of this data, however, varies across subjects as well …