Rare Disease

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New-born screening programs

Published by IRDiRC

Genetic ConditionNewborn Sreening ProgrammePatient InformationRare DiseaseResearch and Drug Development

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.

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EURORDIS’ Community Advisory Boards

Published by IRDiRC

Community Advisory BoardEurordisMedical ResearchOrphan Drug DevelopmentPatient AdvocatePatient InformationPatient OrganisationRare DiseaseResearch and Drug Development

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.

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