Workshops and meetings

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.

Planned activities

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.

Click here to find out more or to register

Activities so far

February 2016 – Workshop on delivery and delivery to the CNS

October 2015 – Exon skipping session at the OTS meeting

June 2015 – ExPRESS summer school

May 2015 – Animal models meeting

April 2015 – Stakeholder workshop at EMA

April 2015  – Presenting scientific work to patients and parents

April 2014 – Chemistry and Toxicology meeting

January 2014 – meeting on Brain Delivery

September 2013 – Exon skipping workshop