Toward therapeutic trials in primary lateral sclerosis.

Erica Scirocco, Matti D Allen, Elisa Giacomelli, Senda Ajroud-Driss, Jinsy Andrews, Sandra Banack, Peter Bede, Michael Benatar, Ken Cheung, Philippe Corcia, Mamede de Carvalho, Lauren Elman, John K Fink, Angela Genge, Orla Hardiman, Matthew Harms, Daragh Heitzman, Grace Jang, Osamu Kano, Matthew C Kiernan, Ikjae Lee, Albert Ludolph, Paul Mehta, Hande Ozdinler, Kourosh Rezania, Paride Schito, Alexander V Sherman, Vincenzo Silani, Eric Sorenson, Martin R Turner, Leonard Van Den Berg, Hiroshi Mitsumoto, Sabrina Paganoni
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Abstract

Primary lateral sclerosis (PLS) is a rare neurodegenerative disorder primarily affecting the upper motor neurons. People living with PLS experience progressive physical and communication disability, which typically evolves slowly over several years. In contrast to amyotrophic lateral sclerosis (ALS), life expectancy is anticipated to be normal. Disease-modifying medications are not available and PLS drug development has been challenging. This review considers recent advances and ongoing initiatives aimed at promoting clinical trial readiness for PLS. Ongoing clinical research efforts include patient registries and biorepositories, natural history studies, outcome measure validation, and biomarker development. These international collaborative efforts are essential for developing the first therapeutic trials for people living with PLS.

原发性侧索硬化的治疗试验。
原发性侧索硬化(PLS)是一种罕见的神经退行性疾病,主要影响上运动神经元。患有PLS的人会经历逐渐的身体和沟通障碍,通常在几年的时间里慢慢发展。与肌萎缩侧索硬化症(ALS)相比,预期寿命正常。疾病改善药物是不可用的,PLS药物开发一直具有挑战性。本综述考虑了旨在促进PLS临床试验准备的最新进展和正在进行的举措。正在进行的临床研究工作包括患者登记和生物储存库、自然历史研究、结果测量验证和生物标志物开发。这些国际合作的努力对于开展针对PLS患者的首批治疗试验至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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