Research Project

Project Title:	ODAP (Orphan Drug Access protocol lumasiran)
Project Type:	Observational Study, Enrolment ongoing
Disease group(s):	Metabolic & stone disorders
Project Summary:	Orphan Drug Access protocol lumasiran: Analysis of a Dutch nation-wide program to improve efficiency and cost-reduction in the prescription of orphan drugs in extreme rare diseases.
Lead principal investigator(s):	Jaap Groothoff, Amsterdam
Co-investigator(s):	Sander Garrelfs, Amsterdam Antonia Bouts, Amsterdam
Project Period:	01/2024 - 01/2029
Sponsors:	Industry