CureDRPLA hosted an in-person research conference in Boston on October 10-11, with 26 attendees. Including researchers from academic institutions in the US, UK, Italy and Japan attended the conference, along with representatives from Vico Therapeutics B.V, Alnylam Pharmaceuticals Inc. and Seelos Therapeutics Inc.
The conference kicked off on October 10 with an opening by Paul and Andrea Compton, who welcomed everyone and gave an overview of our progress over the past year.
Dr. Silvia Prades, DRPLA Research Manager, shared details about CureDRPLA’s ongoing work. Silvia explained our strategy, funding prioritization and the areas of research that interest us. This set the stage for discussions throughout the event. With a room full of experts, we had an excellent opportunity to review our decision-making process and gather their valuable feedback. Silvia also shared the most recent results from the CureDRPLA Global Patient Registry, which currently has 38 participants from eight different countries.
Dr. Jeff Carroll, Scientific Advisor to CureDRPLA, presented the most recent data about a mouse model that mimics the human form of DRPLA and the therapeutic strategies his group has been testing in these DRPLA mice. You can find more details on this research here.
Throughout the first day, we had additional presentations from invited scientists working on DRPLA projects funded by CureDRPLA. We also heard from representatives of pharmaceutical companies regarding their work on other conditions that could be relevant to DRPLA.
On the second day, October 11, we focused on reviewing which therapeutic strategies are relevant for DRPLA and how we can advance research even further. We also had a meeting about the DRPLA Natural History and Biomarkers Study, during which we reviewed the progress of participation at the UK and US study sites.
For CureDRPLA, this conference was an excellent and rewarding experience. We were thrilled to see such high engagement and willingness to collaborate among the attendees in advancing DRPLA research. The feedback we received from those who attended was overwhelmingly positive. We look forward to next year’s conference!