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 …

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 …

Improving clinical trial outcomes in amyotrophic lateral sclerosis

MC Kiernan, S Vucic, K Talbot, CJ McDermott… - Nature Reviews …, 2021 - nature.com
Individuals who are diagnosed with amyotrophic lateral sclerosis (ALS) today face the same
historically intransigent problem that has existed since the initial description of the disease in …

[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 …

Genetics of amyotrophic lateral sclerosis: seeking therapeutic targets in the era of gene therapy

N Suzuki, A Nishiyama, H Warita, M Aoki - Journal of human genetics, 2023 - nature.com
Amyotrophic lateral sclerosis (ALS) is an intractable disease that causes respiratory failure
leading to mortality. The main locus of ALS is motor neurons. The success of antisense …

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 …

New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022

JS Jiang, Y Wang, M Deng - Frontiers in pharmacology, 2022 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder that
primarily affects motor neurons in the brain and spinal cord. In the recent past, there have …

[HTML][HTML] Human stem cell models of neurodegeneration: From basic science of amyotrophic lateral sclerosis to clinical translation

E Giacomelli, BF Vahsen, EL Calder, Y Xu, J Scaber… - Cell Stem Cell, 2022 - cell.com
Neurodegenerative diseases are characterized by progressive cell loss leading to disruption
of the structure and function of the central nervous system. Amyotrophic lateral sclerosis …

[HTML][HTML] iPSC-derived organ-on-a-chip models for personalized human genetics and pharmacogenomics studies

VEJM Palasantzas, I Tamargo-Rubio, K Le, J Slager… - Trends in Genetics, 2023 - cell.com
Genome-wide association studies (GWAS) have now correlated hundreds of genetic
variants with complex genetic diseases and drug efficacy. Functional characterization of …

Comprehensive research on past and future therapeutic strategies devoted to treatment of amyotrophic lateral sclerosis

B Sever, H Ciftci, H DeMirci, H Sever, F Ocak… - International journal of …, 2022 - mdpi.com
Amyotrophic lateral sclerosis (ALS) is a rapidly debilitating fatal neurodegenerative disorder,
causing muscle atrophy and weakness, which leads to paralysis and eventual death. ALS …