The preclinical work on exon skipping for rare diseases is fragmented. Even though many of us work on different diseases, the challenges we face are the same. On the other hand, some of us work on exon skipping for the same disease and we should avoid doing the same work simultaneously.
To harmonize our efforts, and jointly tackle common challenges, regular workshops are organized where participants confidentially discuss hurdles and their latest results.
All our members are involved in this effort.
ExPRESS summer school 2016
A new Expert Patient and Researchers EURORDIS summer school (ExPRESS) will be organized with EURORDIS June 6-10 2016 in Barcelona, Spain. In this summer school Academics and patient representatives will learn about the regulatory processes of drug development and marketing approval in Europe with a strong focus on drug development for rare diseases. This course is highly recommended by Annemieke Aartsma-Rus who attended it in 2013 and co-organized the first ExPRESS summerschool in 2015 with EURORDIS. Anyone who wants to know more about what is required to develop a therapy to clinical implementation will benefit from attending this course.
Deadline for registration is December 31 2015.