We are excited to introduce you to the team that will lead the Australian NPC Disease Foundation (ANPDF) in 2025/26.

Read the full Community Update from Mandos Health here: Mandos Health has shared an October update highlighting exciting progress in Niemann-Pick type C (NPC) research. Their investigational therapy, adrabetadex , continues to show promise, with new data suggesting a substantial survival benefit  in infantile-onset NPC1 and evidence of improved cholesterol trafficking . Their survival study was recognised as an “Abstract of Distinction”  at the American Neurological Associati

Xenpozyme® (olipudase alfa) has been listed on the Life Saving Drugs Program (LSDP) for patients with Acid Sphingomyelinase Deficiency (ASMD).

October is Niemann-Pick Disease Awareness Month and we will be shining a silver light in 2025!

ANPDF is proud to join Neurological Alliance Australia—amplifying our voice for those impacted by Niemann-Pick disease.

At the Australian NPC Disease Foundation (ANPDF) , we know that access to accurate, up-to-date medical guidance can make all the...

Discover how #Resolution4Rare at the 78th WHA is set to revolutionise rare disease care and policy worldwide—offers renewed hope to patients

Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD) and Niemann-Pick type C...

Reflecting on 2024: ANPDF’s progress in advocacy, fundraising, and community support. Looking ahead to a brighter future for NPC in 2025.

“Just go home and make memories” These words have rung in the ears of parents as a doctor drops a life-changing diagnosis of Niemann-Pick...

Mala Singh’s $5,000 donation honours her wish for a cure for Niemann-Pick Disease.

The blood-brain barrier is a roadblock in NPC treatment, but new strategies aim to safely deliver therapies directly to the brain.

As an organisation passionately dedicated to spreading awareness about Niemann-Pick Disease, we are thrilled to delve into the...

Despite Niemann-Pick Disease, Greg’s family remains resilient, providing care, love, and hope for him and others affected by NPC.

15 October 2024 - INPDR, Tyne & Wear England. We are thrilled to share the announcement of a significant development in the fight against...

We invited our favourite Melbourne University volunteer to share his thoughts in this special edition of the NP-C Corner. Check it out!

October is Niemann-Pick Disease Awareness Month and we will be shining a silver light in 2024!

FDA approval of AQNEURSA offers new hope for Australian families with Niemann-Pick disease, promising better treatment and quality of life.

New Hope for NP-C Families: Miplyffa, the first FDA-approved treatment for NPC, offers a promising future for our loved ones. Stay informed!

Limited clinical trial opportunities and a decrease in global trials over the past 5 years means advocacy has never been more crucial.