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

Yang Liu; Xue Xia; Yingfang Chen; Haoying Yang; Xiaochen Chen; Lei Cai; Bingyang Shi

doi:10.1002/exp2.70114

Exploration ›› 2026, Vol. 6 ›› Issue (2) :70114 DOI: 10.1002/exp2.70114

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

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Yang Liu, Xue Xia, Yingfang Chen, Haoying Yang, Xiaochen Chen, Lei Cai, Bingyang Shi. When Rare Is Not Small: Amyotrophic Lateral Sclerosis Initiatives and Therapy. Exploration, 2026, 6 (2) : 70114 DOI:10.1002/exp2.70114

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References

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[1]	The Lancet Global Health, “The Landscape for Rare Diseases in 2024,” The Lancet Global Health 12 (2024): e341, https://doi.org/10.1016/S2214-109X(24)00056-1.

[2]	K. D. Kernohan and K. M. Boycott, “The Expanding Diagnostic Toolbox for Rare Genetic Diseases,” Nature Reviews Genetics 25 (2024): 401–415, https://doi.org/10.1038/s41576-023-00683-w.

[3]	Nature Genetics. “Rare Diseases, Common Challenges,” Nature Genetics 54 (2022): 215–215, https://doi.org/10.1038/s41588-022-01037-8.

[4]	D. Taruscio and W. A. Gahl, “Rare Diseases: Challenges and Opportunities for Research and Public Health,” Nature Reviews Disease Primers 10 (2024): 13, https://doi.org/10.1038/s41572-024-00505-1.

[5]	E. Tambuyzer, B. Vandendriessche, C. P. Austin, et al., “Therapies for Rare Diseases: Therapeutic Modalities, Progress and Challenges Ahead,” Nature Reviews Drug Discovery 19 (2020): 93–111, https://doi.org/10.1038/s41573-019-0049-9.

[6]	World Health Organization. 156th Session, Agenda Item 6. Edited by Executive Board. Geneva: World Health Organization, February 4, 2025. https://apps.who.int/gb/ebwha/pdf_files/EB156/B156_%2814%29-en.pdf.

[7]	S. A. Dugger, A. Platt, and D. B. Goldstein, “Drug Development in the Era of Precision Medicine,” Nature Reviews Drug Discovery 17 (2018): 183–196, https://doi.org/10.1038/nrd.2017.226.

[8]	A. Passaro, M. Al Bakir, E. G. Hamilton, et al., “Cancer Biomarkers: Emerging Trends and Clinical Implications for Personalized Treatment,” Cell 187 (2024): 1617–1635, https://doi.org/10.1016/j.cell.2024.02.041.

[9]	S. H. Kassim, F. Urnov, K. Musunuru, et al., “Platform Solutions for Commercial Challenges to Expanding Patient Access and Making Gene Editing Sustainable,” Nature Biotechnology 43 (2025): 1047–1049, https://doi.org/10.1038/s41587-025-02744-3.

[10]	R. L. Setten, J. J. Rossi, and S.-P. Han, “The Current state and Future Directions of RNAi-Based Therapeutics,” Nature Reviews Drug Discovery 18 (2019): 421–446, https://doi.org/10.1038/s41573-019-0017-4.

[11]	A. Cubillos-Ruiz, T. Guo, A. Sokolovska, et al., “Engineering Living Therapeutics With Synthetic Biology,” Nature Reviews Drug Discovery 20 (2021): 941–960, https://doi.org/10.1038/s41573-021-00285-3.

[12]	B. B. Mendes, J. Conniot, A. Avital, et al., “Nanodelivery of Nucleic Acids,” Nature Reviews Methods Primers 2 (2022): 24, https://doi.org/10.1038/s43586-022-00104-y.

[13]	C. Duan, M. Kang, K. Liu, et al., “RAG-17: A Novel siRNA Conjugate Demonstrating Efficacy in Late-Stage Treatment of SOD1G93A ALS Mice,” preprint, bioRxiv, November 23, 2023, https://doi.org/10.1101/2023.11.23.568255.