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Advancing Antisense-Mediated Exon Skipping for Rare Diseases: A Collaborative Approach

This initiative aims to enhance the clinical application of antisense-mediated exon skipping for rare diseases by uniting all stakeholders, including clinicians, scientists, industry representatives, patients, and regulators. Our objectives include facilitating clinical development, achieving consensus on biochemical outcome measures, and creating new regulatory models for personalized medicine. We will also conduct workshops and exchange visits to foster collaboration and address misconceptions in therapy. Join us to expand the potential of exon skipping for as many patients as possible. For more information, contact Annemieke Aartsma-Rus at a.m.rus@lumc.nl.

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Advancing Antisense-Mediated Exon Skipping for Rare Diseases: A Collaborative Approach

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  1. Networking towards clinical application of antisense-mediated exon skipping Involving all key stakeholders (clinicians, scientists, industry, patients & regulators) • Facilitate the clinical development of antisense-mediated exon skipping for rare diseases • Exploit exon skipping for as many patients as possible Objectives • Achieve consensus biochemical outcome measures • New regulatory models to allow testing of personalized medicine approaches • Foster synergistic work (workshops and key stakeholder meetings and exchange visits) • Address therapeutic misconception by training early stage researchers Current parties involved: Belgium, Denmark, Estonia, France, Germany, Israel, Italy, Malta, Netherlands, Norway, Portugal, Serbia, Spain, Sweden and United Kingdom If you are interested to join, please contact the Chair of this Action Annemieke Aartsma-Rus, a.m.rus@lumc.nl COST Action BM1207

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