Session co-organiser and moderator: Daniël Warmerdam Amsterdam UMC, Netherlands
Panelist: Alvin Luk HuidaGene Therapeutics
Panelist: Cecilia Jimenez-Mallebrera Institut de Recerca Sant Joan de Déu. Hospital Sant Joan de Déu Barcelona and CIBERER
Panelist: Jimi Olaghere Patient Advocate
Panelist: Kevin Holden Synthego
Panelist: Liselotte Wesley Andersen Rare Diseases Denmark
Panelist: Søren Jarl Christensen Parent, Founder and CEO of August Foundation (Sammen for August)