The purposes of Undiagnosed Diseases Programs (UDPs) are to provide patients with an unknown genetic condition a diagnosis and to find the correlation between genotype and phenotype; to share globally the information to facilitate the diagnosis through a matchmaking for finding possible second casesView this resource Bookmark this resource
The purpose of NBS is to detect potentially fatal or disabling conditions in newborns as early as possible and possibly before onset of symptoms. Such detection allows the early treatment which may significantly modify the natural history of the disease and potentially prevent developmental delays, physical disabilities and eventually death.View this resource Bookmark this resource
Patient Community Advisory Boards are groups established and operated by patient advocates. They facilitate discussions in a safe harbour setting and under strict rules, an MOU and CA on the latest developments and challenges related to medical research and procedures in a disease area with the company or body conducting the research. The CAB can cover from preclinical to post marketing topics. The CAB depending on the areas of discussion can invite scientific expert to serve as advisors.View this resource Bookmark this resource
The EUPATI Academy is the only in-depth training program for patient experts so far in EU that lasts 14months.
It includes an on online training program supported with two weeks of F2F training. The Academy is still operated by EPF, providing high quality training in drug development to patient from EMEA.