关于管理下进入协议在即将上市的肌萎缩性脊髓侧索硬化症药物中的作用的范围审查：从脊髓性肌萎缩症案例中汲取经验

IF 2.5 4区医学 Q2 CLINICAL NEUROLOGY

Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration Pub Date : 2024-09-10 DOI:10.1080/21678421.2024.2400522

Beliu García-Parra, Josep M. Guiu, MÓnica Povedano, Pilar Modamio

引用次数: 0

摘要

导言：脊髓性肌萎缩症（SMA）的治疗方案令人鼓舞。然而，肌萎缩性脊髓侧索硬化症（ALS）目前还无法治愈。临床和经济上的不确定性，是治疗肌萎缩性脊髓侧索硬化症（ALS）的关键。

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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A scoping review of the role of managed entry agreements in upcoming drugs for amyotrophic lateral sclerosis: learning from the case of spinal muscular atrophy

Introduction: The therapeutic options for spinal muscular atrophy (SMA) are encouraging. However, there is currently no cure for amyotrophic lateral sclerosis (ALS). The clinical and economic uncer...

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来源期刊

Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration CLINICAL NEUROLOGY-

CiteScore

5.40

自引率

10.70%

发文量

期刊介绍： Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration is an exciting new initiative. It represents a timely expansion of the journal Amyotrophic Lateral Sclerosis in response to the clinical, imaging pathological and genetic overlap between ALS and frontotemporal dementia. The expanded journal provides outstanding coverage of research in a wide range of issues related to motor neuron diseases, especially ALS (Lou Gehrig’s disease) and cognitive decline associated with frontotemporal degeneration. The journal also covers related disorders of the neuroaxis when relevant to these core conditions.