Workshop 1 NET-RARE: Advancements in Personalized Therapies for Rare Diseases
Thursday, February 27 @ 9:30 am–1:30 pm
In this first workshop, we will explore how various IBEC groups and external researchers are using new technologies to develop personalized therapies for rare diseases. The opening seminar features Adriana Gonzalez from IBEC’s “Molecular Imaging for Precision Medicine” group. She will explain how the group studies myotonic dystrophy type I, a rare muscular disease, using nuclear magnetic resonance techniques. Next, Silvia Muro from IBEC’s “Targeted Therapeutics and Nanodevices” group will present recent progress in using targeted nanotechnology to deliver drugs for treating genetic lysosomal disorders. We are also pleased to welcome Frances Platt, professor of Biochemistry and Pharmacology, Department of Pharmacology, University of Oxford (UK). Her group studies fundamental disease mechanisms in lysosomal disorders to identify novel clinical interventions strategies. They use small molecule drugs to target unique steps in the pathogenic cascade and test them as mono-therapies or in combination with other therapies. After a networking break, Belen Perez Dueñas from VHIR’s “Therapeutics and Innovations in Neuropediatrics” group will describe her team’s efforts to address rare neuromuscular disorders through molecular diagnostics and personalized genetic therapies. Finally, Eduard Torrents Serra, leader of IBEC’s “Bacterial Infections: Antimicrobial Therapies” group, will talk about his research on antimicrobial treatments for cystic fibrosis.
Scheduled Program:
09.30-09.40: Welcome and short introduction to the workshop
09.40-10.10: Silvia Blanco, IBEC group “Molecular Imaging for Precision Medicine”
10.10-10.40: Silvia Muro, IBEC group “Targeted therapeutics and nanodevices”
10.40-11.30: International invited speaker: Frances Platt, professor of Biochemistry and Pharmacology, Department of Pharmacology, University of Oxford (UK).
11.30-12.00: Coffee Break and Networking
12.00-12.30: National invited speaker: Belen Perez Dueñas from the VHIR group “Therapeutics and innovations in neuropediatrics”.
12.30-13.00: Eduard Torrents Serra, IBEC group “Bacterial Infections: Antimicrobial Therapies”
13.00-13.10: Remarks and Closing
