Understanding rare melanocortin-4 receptor (MC4R) pathway diseases: from insights to precision medicine
The session brought the journey of a “patient”, Alex (a fictional patient case study), to life, illustrating the need for strong collaboration between paediatric and adult endocrinologists.
During this session, Professor Luca Busetto (University of Padova, Italy), Professor Erica van den Akker (Erasmus University Medical Centre,The Netherlands) and Professor Karine Clément (Pitié Salpêtrière hospital, Sorbonne Université, Inserm France) discussed:
- The impact of living with a rare MC4R pathway disease and the journey to diagnosis
- The role of precision medicine and the most recent data
- The impact of early intervention: Clinical experience with patients suffering from rare MC4R pathway diseases