Wednesday, 9/10/25 – In advance of the 2025 American Neurological Association (ANA) meeting, Mandos Health releases two scientific abstracts with promising data for the treatment of NPC patients with Adrabetadex.
The lead abstract, designated by ANA as an “Abstract of Distinction,” presents data showing a statistically significant survival benefit in patients with infantile-onset NPC who were treated with adrabetadex. A companion abstract offers biomarker evidence indicating that the therapy may reduce neuronal damage by restoring cholesterol trafficking—one of the disease’s core pathological features.
The first abstract, led by Dr. Elizabeth Berry-Kravis of Rush University Medical Center, focuses on survival outcomes in individuals with infantile-onset NPC treated with intrathecal adrabetadex. The data compares these patients with a matched external control group, demonstrating a notable survival benefit that has earned the abstract the conference’s “Abstract of Distinction” honor.
The second abstract, led by Dr. Forbes D. Porter of the National Institutes of Health, details changes in biomarkers associated with neuronal damage. Specifically, the study highlights how improved cholesterol trafficking correlates with reductions in molecular markers of neural injury, providing biological validation that complements the observed survival benefit.
Read an article in Business-News-Today: New hope for Niemann-Pick? Mandos Health drug shows survival benefit ahead of ANA 2025