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William S. Sly

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Burosumab

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CDKL5 deficiency disorder

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http://www.ultragenyx.com/patients/xlh/

Wikipedia: Burosumab

http://www.ultragenyx.com/

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https://www.ultragenyx.com/our-research/pipeline/ux055-for-cdd/

Wikipedia: CDKL5 deficiency disorder