TWO-PRONGED TREATMENT FOR NIEMANN PICK TYPE C DISEASE
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United States of America
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Issued Date -
Apr 10, 2025
app pub date -
May 16, 2023
filing date -
May 16, 2023
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Abstract
Niemann-Pick Disease type C (NPC) is a neurovisceral lysosomal lipid storage disorder that has a wide clinical spectrum. The disclosure provides for the use of oxysterols and optionally, a polypeptide that binds to a Stimulator of Interferon Gene (STING) protein, compositions of the same, and methods using the compounds and composition to treat NPC disease.
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