Evidence map›Paper›PMID 40024955›Full record

SynthesisNeurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology2025

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

Saad Masood, Muhammad Sameer Almas, Syed Saad Ul Hassan, Sameen Tahira, Muhammad Hamza Fiaz, Umm E Aimen Minhas, Hafiz Muhammad Qasim Zafar, Musa Masood

Abstract readSystematic ReviewMeta-Analysis
PubMed Publisher
In one paragraph

Synthesis in Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Article
  2. Review
  3. Dose-dependent effects of Arimoclomol in ALS: insights from a network meta-analysis.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Saad MasoodDepartment of Medicine, Allama Iqbal Medical College, Lahore, Pakistan.ORCID http://orcid.org/0009-0003-9697-524X
Muhammad Sameer AlmasDepartment of Medicine, Allama Iqbal Medical College, Lahore, Pakistan. sameeralmas.1122@gmail.com.ORCID http://orcid.org/0009-0003-1077-7307
Syed Saad Ul HassanDepartment of Medicine, Allama Iqbal Medical College, Lahore, Pakistan.ORCID http://orcid.org/0009-0001-5777-239X
Sameen TahiraDepartment of Medicine, Allama Iqbal Medical College, Lahore, Pakistan.ORCID http://orcid.org/0009-0006-7565-8052
Muhammad Hamza FiazDepartment of Medicine, Allama Iqbal Medical College, Lahore, Pakistan.ORCID http://orcid.org/0009-0004-7952-6714
Umm E Aimen MinhasDepartment of Medicine, Allama Iqbal Medical College, Lahore, Pakistan.ORCID http://orcid.org/0009-0000-8770-1763
Hafiz Muhammad Qasim ZafarDepartment of Medicine, Allama Iqbal Medical College, Lahore, Pakistan.ORCID http://orcid.org/0009-0000-6202-1673
Musa MasoodDepartment of Medicine, Allama Iqbal Medical College, Lahore, Pakistan.ORCID http://orcid.org/0009-0000-3823-4804

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivesAmyotrophic 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.

methodA 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.

resultsA 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.

conclusionThis 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.

Indexed as

Amyotrophic Lateral SclerosisNeuroprotective AgentsHumansHydroxylaminesRandomized Controlled Trials as TopicTreatment OutcomearimoclomolHydroxylaminesNeuroprotective AgentsAmyotrophic lateral sclerosisArimoclomolMotor neuron diseaseNeurodegenerative disorder

Identifiers

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.