Location: Novotel Brussels Airport
Leonardo Da Vincilaan 25
B-1831 Diegem
Morning session:
- 9.00- 9.40: Update by the spoke persons of the scientific board: Goals and Working groups. Sven Garbade & Margreet Wagenmakers
- 9.40-10.00: Activity Update E-IMD Registry. Florian Gleich & Stefan Kölker
- 10.00-10.30:: Update propionic academia project. Kim Chapman
- 10:30 – 11:00: Severity-adjusted evaluation of liver transplantation on health outcome in Urea Cycle Disorders. Roland Posset & Sven Garbade
- 11.00:11:15: Coffee break
- 11.15-12.00: Impact of liver transplantation on health related quality of life in intoxication-
type inborn errors of metabolism and on neurological outcome. Benedetta Greco & Diego
Martinelli - 12.00-12.30: The management and clinical outcomes of pregnancies in females with Urea
Cycle Disorders: a review of the literature and results of an international survey. Proposition
for guidelines. Margreet Wagenmakers - 12.30-13.15 Lunch
Industry session:
- 13.15-14.00: ARG1-D research project and pegzilarginase update. Mattias Rudebeck; Immedica Pharma
- 14.00-14.45: A first-in-human clinical trial of gene editing for the correction of neonatal onset OTC deficiency. George Diaz; iECURE
- 14.45-15.00 Closing remarks