When Rare Is Not Small: Amyotrophic Lateral Sclerosis Initiatives and Therapy

Exploration 2026 Open access

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Authors: Yang Liu, Xue Xia, Yingfang Chen, Haoying Yang, Xiaochen Chen, Lei Cai, Bingyang Shi
DOI: 10.1002/exp2.70114
Cited by: 1
References: 3

Abstract

In the precision-medicine era, rare diseases must not be sidelined in translational infrastructure. The Mr. Cai Lei-led "Ice-Breaking Team" turns an amyotrophic lateral sclerosis patient community into a sustainable ecosystem, realigning philanthropy, data, and research and development to reshape rare-disease pipelines and guide precision therapies, offering a replicable blueprint for rare-disease strategies.

Fields of Study & Subjects

Amyotrophic Lateral Sclerosis Research Biomedical Ethics and Regulation Biotechnology and Related Fields

Publication details

Journal / source
Exploration
Publisher
Wiley
Volume
6
Issue
2
Pages
70114–70114
Publication date
13 February 2026
Language
EN