42 ARDAT members gathered in Barcelona, Spain, for the 5th ARDAT General Assembly Meeting, 28-29th April 2025, hosted by colleagues Asphalion. The two-day meeting began during an unprecedent nationwide power outage, resulting in colleagues using their problem-solving skills to present their work in a somewhat innovative and traditional manner. Despite the challenges, the first day kicked off with a management and ethics update by Dr Rachel Waller and lots of conversation and discussion was had regarding the work being completed across work packages 5, 4 and 3.
Still with no power, WiFi or phone signal the first day concluded with a site-seeing walk to Barcelona Cathedral and luckily enough those that joined the walk, were rewarded with the evening meeting meal as the power started to return to Spain!
The second day begun with virtual attendees now being able to join the meeting with updates provided from work package 2 and Professor Mimoun Azzouz’s recent IND approval from the FDA for SPG47 gene therapy. Dr Sol Ruiz Head of Biologics and ATMP, AEMPS joined remotely to provide an ATMP regulatory update before our panel discussion followed. ARDAT’s PPIE group coordinator Dr Amy Hunter from Genetic Alliance UK, Dr’s Sandra Silva and Christian Brander from GRIN and Dr Francina Munell from Vall d’Hebron University Hospital involved in DMD clinical trials sat on the panel discussing the personal perspectives and the unmet needs of treatment and care for patients and families with rare diseases. The proposed no-cost extension was presented and voted on by consortium members, with final work package breakout sessions providing the opportunity for members to discuss plans for fulfilling remaining tasks and deliverables in the final part of the project.
Overall, despite the challenges the meeting was a great success with lots of discussion and plans for going forward. Thank-you to all involved!
