WORKSHOP: OUTCOME MEASURES FOR GENE THERAPY

09,00 Introductory Remarks on Rare Eye Diseases M. Raponi – L. Buzzonetti
President of the Meeting S. Rizzo H. Dolfuss
Brief introduction on IRDs and role of ERN-EYE from Presidents of the Meeting S. Rizzo H. Dolfuss

09,15 – 12,00 Morning session (Chair – G. Iarossi, H. Dolfuss)

1) Gene therapy in some specific IRDs: where are we now?
2) Analysis of IRD clinical trial experience; what was missing?
9,15 -9,45 RPE65/ USH1B – F. Testa
9,45-10,15 CEP290/USH2A – B. Leroy
10,15 -10,45 RPGR/ ABCA4 related Stargardt disease/CD – A.P. Salvetti
10,45-11 X-linked Retinoschisis – P. Sieving
11-11,15 Holtan RP11 – J Prener
Discussion

13,30-16,00 Afternoon session (Chair – B. Falsini, G.Iarossi)

3) Clinical vision testing (electrophysiology, psychophysics, Multimodal imaging); how to select the most appropriate parameters to evaluate therapy efficacy
13,30-13,50 Electrophysiology – P. Sieving, B. Falsini, G. Iarossi
13,50-14,10 Psychophysics – A. Amato, R. Mastromartino
14,10-14,30 Multimodal imaging – M.P. Salvetti, A. Coppè, G. Gambini

4) 14,45-15,15 ERN-EYE act4red initiative – H. Dolfuss – B. Leroy

5) 15,15-15,45 Patient and regulatory agency perspectives – E. Borgonovi, S. Bologna

Discussion