The DCRT had invited Dr. Timothy Yu from Boston Children’s hospital to give a keynote lecture at the DATS just before the DCRT session on day 2 of the symposium. Tim is the clinician who developed the first N=1 exon skipping antisense oligonucleotide for a patient with Batten disease with a cryptic splicing mutation (milasen). To make it more attractive for him to come all the way to the Netherlands we promised him stroopwafels and a boat tour in Amsterdam. He agreed and arrived in Leiden the day before the start of the DATS and gave a presentation at the LUMC.

In the evening, we went to Amsterdam for a 3-hour boat tour. Dinner was prepared by restaurants along the route and was served on the boat. Very good food by the way, highly recommended if you are looking for an original evening out.

On Thursday morning we all left by train to Nijmegen for the DATS. Marlen Lauffer, Rob Collin (Radboudumc) and Ype Elgersma (ErasmusMC) gave a talk in the DCRT session. Furthermore, we had organized a panel discussion about N=1 regulatory issues with Valentina Cordo, a representative from the European Medicines Agency. DCRT posters were presented by Laurie Kerkhof, Frederique Rotteveel, Bianca Zardetto, Dion den Hertog and Emilio Harris-Mostert (EMC).

posted on June 7, 2023