Monday 2nd July

11.00 – Arrival and registration

11.30 – Welcome
Teresinha Evangelista and Annemieke Aartsma-Rus

11.45 – Brief round of introduction

12.15 – Lunch

Session 1: Translational lifecycle

13.00 – Overview of bench to bedside research
Annemieke Aartsma-Rus

Objective: to outline the different steps of therapy development from idea, to proof of concept studies in model systems, to preclinical optimization studies, clinical trials, drug approval and post marketing surveillance studies

14.00 – Tools of the trade for preclinical research
Volker Straub or Annemieke Aartsma-Rus

Objective: outline different models in preclinical research, their opportunities and limitations, and the need for standardized tests and the TREAT-NMD advisory committee for therapeutics (TACT)

15.30 – Tea/coffee break

16.00 – How the regulatory system works
Violeta Stoyanova-Beninska

Objective: explain the centralized system, how it is organized and how regulators decide whether drugs are eligible for marketing authorization, outline patient involvement in committees – focus on rare diseases

17.00 – Mock TACT review session
Moderated by Volker Straub, Annemieke Aartsma-Rus & Teresinha Evangelista

Objective: learning to have a critical look at preclinical research. In this mock session participants will have been provided with a fictitious TACT application from a company planning a clinical trial in the NMD space. Participants will be split into groups of 8-10 participants to discuss the strengths and limitations and outstanding questions of the application.

18.30 – End of day