2025NPconf: Niemann-Pick Disease, type C at the Crossroads

2025NPconf: The Role of Genetic Counsellors in the Niemann-Pick Diagnosis Pathway

The Importance of the INPDR | Conan Donnelly | International Niemann-Pick Disease Registry

“By the Community, for the Community” | Jackie Imrie | International Niemann-Pick Disease Registry

Making the Most of Data | Conan Donnelly | International Niemann-Pick Disease Registry

What is the INPDR Gateway? | Toni Mathieson | International Niemann-Pick Disease Registry

Telling the Community Story | John Lee Taggart | International Niemann-Pick Disease Registry

2025NPconf: Speech and Cognition in People with Niemann-Pick disease type C

Niemann-Pick disease type C | RTCL.TV

2025NPconf: Together for Niemann-Pick at ANPDF

Niemann-Pick C:The Hidden Disease Behind Slow Learning, Seizures, and Ataxia

Data Quality & the INPDR | Adel Sabet | International Niemann-Pick Disease Registry

An experimental drug gave hope to children with a rare disease. Families plead with FDA for help

Background & History of the INPDR | Toni Mathieson | International Niemann-Pick Disease Registry

Kelly and Aaron's Journey - Navigating Life with an Adult NPC Diagnosis

Exploring the INPDR Gateway | Conan Donnelly | International Niemann-Pick Disease Registry

Introducing the NP GUID Tool – Shaun Bolton

Justin Hopkin – INPDR’s New Chief Scientific Officer

The power of data - Shaun Bolton

2025NPconf: Drug Repurposing & Brain Delivery in Childhood Dementia

Niemann-Pick Disease A Quick Look

2025NPconf: A New Standard of Care for NPC in Australia

2025NPconf: Not Routine Parental Care: Respite and In-Home Support Needs

2025NPconf: Emerging Therapies and Future Directions in NP-C Management

A Rare Disease Webinar: What is a placebo in a rare disease trial?

Alec (21, NPC): NPUK interview takeover

2025NPconf: Legacy Through Creative Art Therapies

2025NPconf: Clinical trials in NPC at the Royal Melbourne Hospital

Driving Research Forward: Using Registry Data to Make a Difference

A 13-year-old's journey with a rare disease

What is ASMD? A Rare Genetic disorder also known as NiemannPick Type A/ Type B

Enfermedad de Niemann-Pick #niemannpick #niemannpicktypec #enfermedad #medicina

19 is such an huge milestone when you have a degenerative disease #birthday #milestone #niemannpick

Value of the Clinician Reported Database (CRD) | Shaun Bolton | the INPDR

Watch Liam on News3!

Biochemistry Chapter 2 | Lysosomal Storage Diseases Explained | High-Yield Step 1 Review