1. Empowerment of patients: lessons from the rare diseases community;Aymé;Lancet,2008
2. The added value of patients’ organizations;European patients Forum,2017
3. A journey of hope: lessons learned from studies on rare diseases and orphan drugs;Wästfelt;J Intern Med,2006
4. Pharmacology and drug development in rare diseases: the attractiveness and expertise of the French medical pharmacology;Micallef;Fundam Clin Pharmacol,2017
5. Patients’priorities and needs for rare disease research 2014–2020;Eurordis,2011