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GEMALS:一种有前景的肌萎缩侧索硬化症治疗方法。

GEMALS: A promising therapy for amyotrophic lateral sclerosis.

作者信息

Geffard Michel, Mangas Arturo, Bedat Denis, Coveñas Rafael

机构信息

Research Department, Institute for Development of Research on Human Pathology and Therapeutics, 33400 Talence, France.

Gemacbio, Research Department, Lieu dit Berganton, 33127 Saint Jean d'Illac, France.

出版信息

Exp Ther Med. 2018 Apr;15(4):3203-3210. doi: 10.3892/etm.2018.5868. Epub 2018 Feb 14.

DOI:10.3892/etm.2018.5868
PMID:29545836
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5841048/
Abstract

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that currently has no cure. At present, the only approved treatment for ALS is Riluzole, a glutamate release blocker that improves life expectancy by 3-6 months. ALS-Endotherapia (GEMALS) is a novel therapeutic approach to treat ALS and the aim of the present study was to investigate the potential beneficial effects of this novel treatment. A total of 31 patients with ALS were assessed in the current study. Deceleration of the disease was observed in 83.87% (P<0.0001) of patients and mean life expectancy was increased by 38 months. Motor functions, including breathing, walking, salivation, speech, swallowing and writing, were also improved in patients treated with GEMALS. The results of the present study demonstrate that long-term treatment with GEMALS has a curative effect in patients with ALS. Furthermore, the overall effectiveness of GEMALS was assessed using the ALS Assessment Questionnaire. The score improvement was 76.2 and 100% for men and women, respectively (P<0.0001), compared with the worldwide reference score. The present study provides a promising basis for the use of GEMALS as a therapeutic treatment for patients with ALS; however, these results must be confirmed in a double-blinded and randomized clinical trial.

摘要

肌萎缩侧索硬化症(ALS)是一种目前无法治愈的神经退行性疾病。目前,唯一被批准用于治疗ALS的药物是利鲁唑,一种谷氨酸释放阻滞剂,可将预期寿命延长3至6个月。ALS-Endotherapia(GEMALS)是一种治疗ALS的新型疗法,本研究的目的是调查这种新疗法的潜在益处。本研究共评估了31例ALS患者。83.87%(P<0.0001)的患者病情出现缓解,平均预期寿命延长了38个月。接受GEMALS治疗的患者的运动功能,包括呼吸、行走、流涎、言语、吞咽和书写,也得到了改善。本研究结果表明,长期使用GEMALS对ALS患者具有治疗效果。此外,使用ALS评估问卷评估了GEMALS的总体有效性。与全球参考分数相比,男性和女性的分数改善分别为76.2%和100%(P<0.0001)。本研究为将GEMALS用作ALS患者的治疗方法提供了有前景的依据;然而,这些结果必须在双盲随机临床试验中得到证实。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4d5f/5841048/2d91e09f93ab/etm-15-04-3203-g03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4d5f/5841048/b0c81135385d/etm-15-04-3203-g00.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4d5f/5841048/9c327acb9014/etm-15-04-3203-g01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4d5f/5841048/cc06cd41b0dd/etm-15-04-3203-g02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4d5f/5841048/2d91e09f93ab/etm-15-04-3203-g03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4d5f/5841048/b0c81135385d/etm-15-04-3203-g00.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4d5f/5841048/9c327acb9014/etm-15-04-3203-g01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4d5f/5841048/cc06cd41b0dd/etm-15-04-3203-g02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4d5f/5841048/2d91e09f93ab/etm-15-04-3203-g03.jpg

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