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Current State and Future Directions in the Therapy of ALS
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. As the exact underlying causative pathological pathway is unclear and potenti...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10252394/ https://www.ncbi.nlm.nih.gov/pubmed/37296644 http://dx.doi.org/10.3390/cells12111523 |
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author | Tzeplaeff, Laura Wilfling, Sibylle Requardt, Maria Viktoria Herdick, Meret |
author_facet | Tzeplaeff, Laura Wilfling, Sibylle Requardt, Maria Viktoria Herdick, Meret |
author_sort | Tzeplaeff, Laura |
collection | PubMed |
description | Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. As the exact underlying causative pathological pathway is unclear and potentially diverse, finding a suitable therapy to slow down or possibly stop disease progression remains challenging. Varying by country Riluzole, Edaravone, and Sodium phenylbutyrate/Taurursodiol are the only drugs currently approved in ALS treatment for their moderate effect on disease progression. Even though curative treatment options, able to prevent or stop disease progression, are still unknown, recent breakthroughs, especially in the field of targeting genetic disease forms, raise hope for improved care and therapy for ALS patients. In this review, we aim to summarize the current state of ALS therapy, including medication as well as supportive therapy, and discuss the ongoing developments and prospects in the field. Furthermore, we highlight the rationale behind the intense research on biomarkers and genetic testing as a feasible way to improve the classification of ALS patients towards personalized medicine. |
format | Online Article Text |
id | pubmed-10252394 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-102523942023-06-10 Current State and Future Directions in the Therapy of ALS Tzeplaeff, Laura Wilfling, Sibylle Requardt, Maria Viktoria Herdick, Meret Cells Review Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. As the exact underlying causative pathological pathway is unclear and potentially diverse, finding a suitable therapy to slow down or possibly stop disease progression remains challenging. Varying by country Riluzole, Edaravone, and Sodium phenylbutyrate/Taurursodiol are the only drugs currently approved in ALS treatment for their moderate effect on disease progression. Even though curative treatment options, able to prevent or stop disease progression, are still unknown, recent breakthroughs, especially in the field of targeting genetic disease forms, raise hope for improved care and therapy for ALS patients. In this review, we aim to summarize the current state of ALS therapy, including medication as well as supportive therapy, and discuss the ongoing developments and prospects in the field. Furthermore, we highlight the rationale behind the intense research on biomarkers and genetic testing as a feasible way to improve the classification of ALS patients towards personalized medicine. MDPI 2023-05-31 /pmc/articles/PMC10252394/ /pubmed/37296644 http://dx.doi.org/10.3390/cells12111523 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Tzeplaeff, Laura Wilfling, Sibylle Requardt, Maria Viktoria Herdick, Meret Current State and Future Directions in the Therapy of ALS |
title | Current State and Future Directions in the Therapy of ALS |
title_full | Current State and Future Directions in the Therapy of ALS |
title_fullStr | Current State and Future Directions in the Therapy of ALS |
title_full_unstemmed | Current State and Future Directions in the Therapy of ALS |
title_short | Current State and Future Directions in the Therapy of ALS |
title_sort | current state and future directions in the therapy of als |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10252394/ https://www.ncbi.nlm.nih.gov/pubmed/37296644 http://dx.doi.org/10.3390/cells12111523 |
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