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عنوان فارسی مقاله:

خواص فارمالوژیک داروهای میکرونوروتروفین توسعه یافته برای درمان اسکلروزیس جانبی آمیوتروفیک


عنوان انگلیسی مقاله:

Pharmacological properties of microneurotrophin drugs developed for treatment of amyotrophic lateral sclerosis


سال انتشار : 2016



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مقدمه انگلیسی مقاله:

1. Introduction

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease (NDD) of adults first described in Western medicine by Charcot in the late 19th century [1]. ALS is usually rapidly fatal with death, in the absence of artificial ventilation, due to respiratory failure within 2–5 years of diagnosis. Pathologically, ALS develops from the accelerated dysfunction and death of upper and lower motor neurons, leading to spasticity, weakness, muscle atrophy, dysphagia and ventilatory failure. Most ALS arises ‘‘sporadically” without known autosomal mutations (sporadic ALS, sALS), with 5–10% of cases arising in families (familial ALS, fALS) from point mutations in at least 20 known genes or hexanucleotide expansions in the non-coding sequence between alternate 50 exons in transcripts from C9orf72 [2,3]. This gene is associated with 9p-linked ALS and frontotemporal dementia (FTD [3,4]). Abnormally expanded C9orf72 is the most common known cause of fALS (40%), is found in up to 11% of patients with sALS, and appears to interfere with RNA processing, export from the nucleus and protein translation [2–4]. There are no meaningful disease-altering therapies for sALS in spite of multiple large drug trials to determine efficacy. There are many potential reasons for these failures, including the possibility that sALS is a syndrome, like in many malignancies, where multiple different molecular genetic etiologies can yield similar clinical phenotypes. If true, then a search for a single universal therapy targeting one gene or pathway has limited chances for success, and a better approach could include drugs with pleiotropic effects on multiple neuronal survival pathways.



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کلمات کلیدی:

Evidence-Based Drug Treatment in Amyotrophic Lateral Sclerosis and ... https://www.ncbi.nlm.nih.gov › NCBI › Literature › PubMed Central (PMC) by AC Ludolph - ‎2009 - ‎Cited by 22 - ‎Related articles We review treatment development in motor neuron disease and discuss the ... Keywords: amyotrophic lateral sclerosis, evidence-based therapy, clinical trials. The pre-clinical discovery of Amyotrophic Lateral Sclerosis Drugs https://www.ncbi.nlm.nih.gov › NCBI › Literature › PubMed Central (PMC) by MA Glicksman - ‎2011 - ‎Cited by 16 - ‎Related articles Only one therapeutic drug, Riluzole, has been approved for ALS and that drug ..... ALS Therapy Development Institute (ALS-TDI) has a number of compounds ... Potential therapeutic drugs and methods for the treatment of ... https://www.ncbi.nlm.nih.gov/pubmed/24934355 by G Yacila - ‎2014 - ‎Cited by 15 - ‎Related articles Curr Med Chem. 2014;21(31):3583-93. Potential therapeutic drugs and methods for the treatment of amyotrophic lateral sclerosis. Yacila G, Sari Y(1). New Treatments in Amyotrophic Lateral Sclerosis - Nature www.nature.com › Journal home › Archive › Nppr Hot Topics For the past 15 years, the field of amyotrophic lateral sclerosis (ALS) pathophysiology and drug development has largely been dominated by understanding the ... What is ALS | ALS Therapy Development Institute www.als.net/what-is-als/ This urgent unmet medical need for effective treatments for this devastating and fatal disease is the basis for the research and drug development effort at the ... Disease modifying treatment of amyotrophic lateral sclerosis - UpToDate https://www.uptodate.com/.../disease-modifying-treatment-of-amyotrophic-lateral-scle... Feb 28, 2017 - Amyotrophic lateral sclerosis (ALS), first described by Charcot in the nineteenth ... Riluzole is the only drug to have any impact on survival in ALS [2]. .... Advances in the Development of Disease-Modifying Treatments for ... Searches related to drugs developed for treatment of amyotrophic lateral sclerosis prognosis for als als treatment breakthrough als prevention als treatments new als treatment als disease life expectancy als medication riluzole als treatment 2016