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I acknowledge extensive discussions with the rare disease parents and foundations led by L. Sames, A. Moore and J. Wood, and from discussions with researchers P. Dickson, T.-F. Chou, and C. Heldermon. The Sanfilippo type D enzyme replacement study is funded by NIH NINDS 2R42NS089061-02.
S.E. is a co-founder and employee of Collaborations Pharmaceuticals, Inc. and Phoenix Nest and consults for the Hereditary Neuropathy Foundation.
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Ekins, S. Industrializing rare disease therapy discovery and development. Nat Biotechnol 35, 117–118 (2017). https://doi.org/10.1038/nbt.3787
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