Ataxia UK has a collaborative partnership with CureDRPLA, a US-based non-profit organization that funds scientific projects to advance research for the rare ataxia Dentatorubral-pallidoluysian atrophy (DRPLA). Both Dr Julie Greenfield and Dr Silvia Prades from Ataxia UK are involved in a collaboration to advance DRPLA research and support CureDRPLA in their activities.
CureDRPLA hosted an in-person research conference in Boston on the 10th and 11th of October 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.
Dr Silvia Prades, DRPLA Research Manager, gave a presentation about CureDRPLA’s ongoing work. 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 with DRPLA 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 the mice.
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.
We spent some time 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 study that is currently recruiting participants to better understand DRPLA progression over time. Find out more about this study here.
For CureDRPLA and Ataxia UK, 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.