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Mandos Health Community Update | October, 2025

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Read the full Community Update from Mandos Health here:



Mandos Health has shared an October update highlighting exciting progress in Niemann-Pick type C (NPC) research. Their investigational therapy, adrabetadex, continues to show promise, with new data suggesting a substantial survival benefit in infantile-onset NPC1 and evidence of improved cholesterol trafficking.


Their survival study was recognised as an “Abstract of Distinction” at the American Neurological Association Annual Meeting — a significant milestone as Mandos moves toward a New Drug Application submission.


Mandos Health also reaffirmed its commitment to the Expanded Access Program, ensuring continued access for families.


It was great to meet the Mandos team in person at the INPDA Summit in Argentina, where their dedication to the NPC community was clear.

To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere."


Ways to support NP-C in Australia:

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