Stakeholder communication

For many rare diseases there is an unmet medical need and patients and family members are eagerly awaiting a therapy. At the same time, researchers often oversell results of early phase studies in cells and animal models. This results in unrealistic expectations in the patient communities, regarding what a therapeutic approach can achieve, the timeframe is needed to develop the approach and the potential challenges that may prevent the approach from ever being developed as a registered drug.

The aim of the stakeholder communication (SC) working group is to train early stage researchers in how to present their work in a clear and unbiased way to lay people and patients and also to disseminate the results of the COST Action to the public.

Working group leaders

Annemieke Aartsma-Rus - Netherlands
Mirjam Franken-Verbeek - Netherlands

Other members

Tom Delanoy – Belgium
James Barrow – United Kingdom

Future activities

Opportunity to present exon skipping work

A COST sponsored session on exon skipping is planned for the annual meeting of the Oligonucleotide Therapeutics Society (October 11-15, Leiden, the Netherlands). This session will feature many short talks from selected abstracts and early stage researchers are encouraged to submit abstracts (check the OTS website for updates)

Past activities

Training School

An interactive workshop where a small group of early stage researchers were trained in how to present their research work in a clear and understandable way to lay people and patients/parents will take place in April 2015 in Leiden. The training school was attended by 16 early stage researchers.


Annemieke Aartsma-Rus will present the activities and achievements of the network on February 17th in London during the RNA Therapeutics meeting organized by SMI.