Registry services
Resources
- In this section:
- Registry services
- Resources
- Publications
- Press Releases
Over recent years we’ve been lucky enough to share work at conferences all over the world – meeting and collaborating with so many inspiring patients, families, researchers, scientists, expert clinicians and students along the way. You can find associated scientific posters and other resources developed as part of these meetings below.
- ASMD Niemann-Pick Disease - Guidelines - 2023
- Niemann-Pick Disease (NPC) - Guidelines - 2023
- INPDR Gateway Press Release - 1st June 2023
- ECRD 2020: A Global Gateway to a Lasting Legacy
- WORLD 2020: Characteristics of NPC and ASMD patients
- WORLD 2020: The characteristics of NPC and ASMD patients platform presentation
- WORLD 2020: A beacon for rare diseases
- SSIEM 2019: Building Bridges for the future
- WORLD 2019: Challenges of regulatory requirements for patient registries in different countries
- WORLD 2018: A new model of patient-empowered data ownership and management