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Safety and efficacy of arimoclomol in amyotrophic lateral sclerosis: a systematic review and meta-analysis.

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Title: Safety and efficacy of arimoclomol in amyotrophic lateral sclerosis: a systematic review and meta-analysis.
Authors: Masood, Saad (AUTHOR), Almas, Muhammad Sameer (AUTHOR), Hassan, Syed Saad Ul (AUTHOR), Tahira, Sameen (AUTHOR), Fiaz, Muhammad Hamza (AUTHOR), Minhas, Umm E Aimen (AUTHOR), Zafar, Hafiz Muhammad Qasim (AUTHOR), Masood, Musa (AUTHOR)
Source: Neurological Sciences. Jul2025, Vol. 46 Issue 7, p2985-2994. 10p.
Subjects: Motor neuron diseases, Amyotrophic lateral sclerosis, Muscle weakness, Statistical significance, Medical sciences
Abstract: Objectives: Amyotrophic Lateral Sclerosis (ALS) is a debilitating motor neuron disorder characterized by muscle weakness, atrophy, and spasticity. This meta-analysis aims to assess the safety and efficacy of Arimoclomol in patients with ALS. Method: A comprehensive literature search was conducted on 3 databases to discover articles published up to August 2024. Included studies were randomized controlled trials (RCTs). Data was analysed using Review Manager (v5.4). Cochrane Risk of Bias-2 (RoB-2) was adopted to assess the quality of RCTs. Results: A total of 359 patients were analysed, with 239 individuals in the Arimoclomol group and 120 individuals in the placebo group. The pooled analysis of the primary outcome, change in Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score from baseline, did not demonstrate a statistically significant difference favoring the Arimoclomol group (MD = 0.4495; 95% CI: -0.39, 1.27; p = 0.30). Similarly, secondary outcomes, including the Combined Assessment of Function and Survival (CAFS) rank score (MD = 1.00; 95% CI: -2.68, 4.67; p = 0.60), increase in transaminases (RR = 1.05; 95% CI: 0.19, 5.70; p = 0.95), mortality rate (RR = 0.86; 95% CI: 0.55, 1.34; p = 0.50), and adverse events (RR = 0.86; 95% CI: 0.55, 1.34; p = 0.50), showed no statistically significant differences between the groups. Conclusion: This study does not conclusively demonstrate that Arimoclomol has beneficial effects on ALS patients' physical functionality but shows promise for safety. Further clinical trials are needed to explore the neuroprotective effects of Arimoclomol in the treatment of ALS. [ABSTRACT FROM AUTHOR]
Copyright of Neurological Sciences is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)
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  Data: Safety and efficacy of arimoclomol in amyotrophic lateral sclerosis: a systematic review and meta-analysis.
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  Data: <searchLink fieldCode="AR" term="%22Masood%2C+Saad%22">Masood, Saad</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Almas%2C+Muhammad+Sameer%22">Almas, Muhammad Sameer</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Hassan%2C+Syed+Saad+Ul%22">Hassan, Syed Saad Ul</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Tahira%2C+Sameen%22">Tahira, Sameen</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Fiaz%2C+Muhammad+Hamza%22">Fiaz, Muhammad Hamza</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Minhas%2C+Umm+E+Aimen%22">Minhas, Umm E Aimen</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Zafar%2C+Hafiz+Muhammad+Qasim%22">Zafar, Hafiz Muhammad Qasim</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Masood%2C+Musa%22">Masood, Musa</searchLink> (AUTHOR)
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  Data: <searchLink fieldCode="JN" term="%22Neurological+Sciences%22">Neurological Sciences</searchLink>. Jul2025, Vol. 46 Issue 7, p2985-2994. 10p.
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  Data: <searchLink fieldCode="DE" term="%22Motor+neuron+diseases%22">Motor neuron diseases</searchLink><br /><searchLink fieldCode="DE" term="%22Amyotrophic+lateral+sclerosis%22">Amyotrophic lateral sclerosis</searchLink><br /><searchLink fieldCode="DE" term="%22Muscle+weakness%22">Muscle weakness</searchLink><br /><searchLink fieldCode="DE" term="%22Statistical+significance%22">Statistical significance</searchLink><br /><searchLink fieldCode="DE" term="%22Medical+sciences%22">Medical sciences</searchLink>
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  Data: Objectives: Amyotrophic Lateral Sclerosis (ALS) is a debilitating motor neuron disorder characterized by muscle weakness, atrophy, and spasticity. This meta-analysis aims to assess the safety and efficacy of Arimoclomol in patients with ALS. Method: A comprehensive literature search was conducted on 3 databases to discover articles published up to August 2024. Included studies were randomized controlled trials (RCTs). Data was analysed using Review Manager (v5.4). Cochrane Risk of Bias-2 (RoB-2) was adopted to assess the quality of RCTs. Results: A total of 359 patients were analysed, with 239 individuals in the Arimoclomol group and 120 individuals in the placebo group. The pooled analysis of the primary outcome, change in Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score from baseline, did not demonstrate a statistically significant difference favoring the Arimoclomol group (MD = 0.4495; 95% CI: -0.39, 1.27; p = 0.30). Similarly, secondary outcomes, including the Combined Assessment of Function and Survival (CAFS) rank score (MD = 1.00; 95% CI: -2.68, 4.67; p = 0.60), increase in transaminases (RR = 1.05; 95% CI: 0.19, 5.70; p = 0.95), mortality rate (RR = 0.86; 95% CI: 0.55, 1.34; p = 0.50), and adverse events (RR = 0.86; 95% CI: 0.55, 1.34; p = 0.50), showed no statistically significant differences between the groups. Conclusion: This study does not conclusively demonstrate that Arimoclomol has beneficial effects on ALS patients' physical functionality but shows promise for safety. Further clinical trials are needed to explore the neuroprotective effects of Arimoclomol in the treatment of ALS. [ABSTRACT FROM AUTHOR]
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  Data: <i>Copyright of Neurological Sciences is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
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              Text: Jul2025
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