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 Martin Wabitsch (University Medical Center, Ulm - Germany), Dr Elizabeth Forsythe (Great Ormond Street Hospital, Guy's Hospital and St Thomas' Hospital, London - United Kingdom) and Professor Christine Poitou (Sorbonne Universités, Paris - 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