July 9, 2026 Apertura Gene Therapy Announces Cooperative Research and Development Agreement with Institutes of the NIH to Test Investigational Gene Therapy for Niemann-Pick Disease Type C1 — Cooperative Research and Development Agreement (CRADA) will utilize Apertura’s TfR1 CapX™ Capsid — — Ara Parseghian Medical Research Fund, a non-profit organization dedicated to finding a treatment …
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NP-C
9th Annual DART TO THE FINISH Charity Walk * Sat. * Oct. 3, 2026
Gather your friends and family and join is for another invigorating 2-mile walk with beautiful views of Long Island Sound around Tod’s Point in Old Greenwich, and help us raise much needed funds to find a cure for Niemann-Pick type C disease. No beach pass, no problem. DART has you covered! The morning always makes …
Mandos Submits New Drug Application to U.S. FDA for Adrabetadex in Infantile-Onset NPC!
• 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 …