Major research advances in amyotrophic lateral sclerosis in 2021

In recent clinical trials of pharmacological treatments, differential benefits have been noted in specific patient subgroups, often with more favourable results in patients with fast progressing amyotrophic lateral sclerosis. [...]awareness of disease heterogeneity is increasing. [...]there is a gro...

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Bibliographic Details
Published inLancet neurology Vol. 21; no. 1; pp. 14 - 15
Main Author Petri, Susanne
Format Journal Article
LanguageEnglish
Published England Elsevier Ltd 01.01.2022
Elsevier Limited
Subjects
Amyotrophic lateral sclerosis
Amyotrophic Lateral Sclerosis - therapy
Apoptosis
Biomarkers
Clinical trials
Drug therapy
Epigenetics
Humans
Neurodegeneration
Neuromodulation
p53 Protein
Patients
Proline
R&D
Research & development
Risk factors
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Summary:In recent clinical trials of pharmacological treatments, differential benefits have been noted in specific patient subgroups, often with more favourable results in patients with fast progressing amyotrophic lateral sclerosis. [...]awareness of disease heterogeneity is increasing. [...]there is a growing need to test novel pharmacological and genetic treatment approaches over a shorter trial period. A study by Maor-Nof and colleagues10 elucidated how the dipeptide protein poly-proline-arginine, which is generated from the repeat expansion, induces apoptotic pathways and, ultimately, neurodegeneration by epigenetic modulation of gene transcription via the transcription factor TP53, thereby indicating nucleoporin p54 ablation as a novel therapeutic strategy.10 Overall, amyotrophic lateral sclerosis research in 2021 has resulted in increasing knowledge on risk factors and mechanisms, and in advances in biomarker research and trial design.
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ISSN:1474-4422
1474-4465
DOI:10.1016/S1474-4422(21)00420-8