Children living with Neimann-Pick disease type C

WHO WE ARE

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We are a purpose-driven company focused on bringing life-changing therapeutics to people living with rare diseases.
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Adult living with Niemann-Pick disease type C
Adult living with Niemann-Pick disease type C

WHAT WE DO

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We address the unmet needs of patients living with rare diseases through leading scientific advances and the collective strength of passionate people.

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Adult living with Niemann-Pick disease type C
Adult living with Niemann-Pick disease type C

WHY WE DO IT

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We are zealous in our pursuit of developing and delivering therapies that address significant unmet needs because patients and their care partners don’t have a moment to lose.

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Child living with Niemann-Pick disease type C
Child living with Niemann-Pick disease type C

Our Products

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Our mission is to bring life-changing therapeutics to people living with rare diseases.
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