Satellite Meeting
29th March 2025
Raising Awareness in innovative therapies in IMD
Saturday, 29th March
| 09:30 |
Secretariat opening |
| 10:00 |
Satellite Meeting Opening – Welcome Address on behalf of the SPDM |
| 10:10 |
RNAi - revolutionary new treatment approach for acute hepatic porphyrias: Givosiran (Alnylam) Daniel Costa Gomes, Lisbon, PT |
| 10:40 |
Cerliponase alfa: state of the art in CLN2 (BioMarin) Sofia Quinta, Lisbon, PT |
| 11:10 |
Coffee Break |
| 11:40 |
Challenges in the diagnosis of LAL-D (Alexion) Patrício Aguiar, Lisbon, PT |
| 12:10 |
Avalglucosidase alfa: what is the impact on the Pompe disease patients’ quality of life? (Sanofi) Miguel Santos, Lisbon, PT |
| 12:10 |
Discussion> |
