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Welcome to the ANPDF blog, where we share the latest updates, stories, and breakthroughs in the fight against Niemann-Pick Disease. Join us as we raise awareness, support families, and drive research forward.

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NP-C Corner: Unpacking the Blood-Brain Barrier – A Key to NPC Treatment

Ellie Van Velsen

Nov 83 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.

69 views

Second treatment approved by FDA for Niemann-Pick disease, type C: Introducing AQNEURSA

ANPDF

Sep 254 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.

94 views

A New Hope for NP-C Families: Introducing Miplyffa

ANPDF

Sep 212 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!

105 views

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