Registry services
Publications
- In this section:
- Registry services
- Resources
- Publications
- SMPD1 Mutation Update: Database and Comprehensive Analysis of Published and Novel Variants
- The International Niemann-Pick Disease Registry (INPDR) – A new model of patient-empowered data ownership and management
- Challenges of regulatory requirements for patient registries in different countries
- Consensus clinical management guidelines for Niemann-Pick disease type C
