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Ellie Van Velsen
Nov 8, 20243 min read
NP-C Corner: Unpacking the Blood-Brain Barrier – A Key to NPC Treatment
The blood-brain barrier is a roadblock in NPC treatment, but new strategies aim to safely deliver therapies directly to the brain.
81 views
ANPDF
Sep 25, 20244 min read
Second treatment approved by FDA for Niemann-Pick disease, type C: Introducing AQNEURSA
FDA approval of AQNEURSA offers new hope for Australian families with Niemann-Pick disease, promising better treatment and quality of life.
107 views
ANPDF
Sep 21, 20242 min read
A New Hope for NP-C Families: Introducing Miplyffa
New Hope for NP-C Families: Miplyffa, the first FDA-approved treatment for NPC, offers a promising future for our loved ones. Stay informed!
108 views