Sunday 29th Programme
Session 5: Accelerating on the bumpy road to therapy
The bumpy road to therapy - Bert Smeets
8:30 - 8:50
Triple AAV gene therapy to express full-length LAMA2 - Marc Güell
Mesenchymal-derived cells and tissues as therapeutic targets for MDC1A - Maria Pallarés Masmitjà
8:50 - 9:10
9:10 - 9:30
3D Engineered Muscle Tissues Enable Longitudinal Muscle Functional Studies for Neuromuscular Disease - Mohammad Khoonkari, Hamed Ghazizadeh (CuriBio)
9:30 - 9:50
Linker-based gene therapy for LAMA2-RD - Markus Rüegg
9:50 - 10:10
Coffee break
10:10 - 10:30
The power of patients - Jasmin Barman-Aksözen
10:30 - 11:00
A role for patient advocacy groups -Gustavo Dziewczapolski, Bram Verbrugge
11:00 - 11:20
A role for rare disease research foundations/regulatory authorities - Giorgia Crimi, Federica Miotto (Fondazione Telethon, Italy)
11:20 - 11:40
iPSC models for LAMA2-RD -Józef Dulak
11:40 - 12:00
Clinical Evaluation of LAMA1 Upregulation: Translating Decades of Research to Meaningful Outcomes for LAMA2-RD Patients - Simon Moore (Modalis)
12:00 -12:20
Joining forces towards an accessible and affordable therapy - TBC/Bert Smeets
12:20 - 12:50
Closure of the meeting - Haluk Topaloğlu,Bert Smeets, and Bram Verbrugge
12:50 - 13:00
Closing lunch
13:00 - 14:00