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Trends in drug development for amyotrophic lateral sclerosis

  1. Selma Kraft

    1. US Food and Drug Administration, Silver Spring, MD, USA.

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  2. Catherine Mease

    1. US Food and Drug Administration, Silver Spring, MD, USA.

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  3. Devanand Jillapalli

    1. US Food and Drug Administration, Silver Spring, MD, USA.

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  4. Lewis J. Fermaglich

    1. US Food and Drug Administration, Silver Spring, MD, USA.

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  5. Kathleen L. Miller

    1. US Food and Drug Administration, Silver Spring, MD, USA.

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Amyotrophic lateral sclerosis (ALS) is a severe progressive, neurodegenerative disease. It is also rare, and has been the focus of substantial efforts to develop new therapies in recent years. As such, ALS could provide a useful example for exploring the potential impact of financial incentives to encourage the development of drugs for rare diseases, which were introduced in the United States 40 years ago with the passage of the Orphan Drug Act.

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Nature Reviews Drug Discovery 23, 99-100 (2024)

doi: https://doi.org/10.1038/d41573-023-00199-2

Supplementary Information

  1. Supplementary Table 1

Competing Interests

The authors declare no competing interests. This article reflects the views of the authors and should not be construed to represent the FDA’s views or policies.

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