Gagnon, Cynthia

Purpose

The specific objectives of this funding opportunity are to: • Enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with expected impact to use the results in the future for benefit of patients. • Conduct efficient, innovative and high-quality natural history studies which will facilitate the understanding of the progression of rare diseases - or group of disorders - throughout the lifespan of a patient. The goal of these studies is to collect and analyse comprehensive patient data to define targets for future therapies, taking into consideration innovation, safety, and efficacy.