Treatments fashioned specifically for rare diseases are necessarily very expensive. A fully satisfactory solution to this problem has not yet been found.
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Beutler, E. The cost of treating Gaucher disease. Nat Med 2, 523–524 (1996). https://doi.org/10.1038/nm0596-523
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DOI: https://doi.org/10.1038/nm0596-523
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