• Comprehensive submission package includes evidence of substantially improved survival among adrabetadex-treated patients with infantile-onset Niemann Pick disease type C (NPC), intended to serve as a single adequate and well-controlled study, and confirmatory evidence of biomarkers and nonclinical data demonstrating effects on the underlying pathology • Priority Review was requested on the basis that the …
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niemann-pick type c
Adrabetadex (Cyclodextrin) receives FDA Breakthrough Therapy Designation for Individuals with Infantile-Onset NPC
Breakthrough Therapy Designation is based on FDA’s review of survival analyses comparing adrabetadex-treated patients with external controls and marks a significant regulatory milestone ahead of an New Drug Apllication submission. THOUSAND OAKS, Calif.– December 9, 2025 – Beren Therapeutics P.B.C.®, a Public Benefit Corporation and parent company of Mandos LLC®, today announced that Mandos received Breakthrough Therapy Designation …
2025 Annual DART to the Finish Charity Walk Raises Over $160,000
Dana’s Angels Research Trust (DART), the nonprofit organization dedicated to funding medical research, education and treatment of the rare childhood disease Niemann-Pick Type C (NPC), often referred to as “childhood Alzheimer’s,” had the most perfect fall day for its 8th annual DART to the Finish Charity Walk, held Saturday, October 4, 2025 at Greenwich Point …