Last week, the 5th edition of Duchenne Patient Academy has taken place virtually. More than 75 Duchenne and Becker MD patient advocates from over 40 countries actively participated in the 5-day training. The goal of this Academy is to educate and strengthen the global Duchenne and Becker Muscular Dystrophy community. Duchenne Patient Academy is a collaboration between the World Duchenne Organization and the Duchenne Data Foundation.
Each day focused on a specific topic, based on questions from the community. George Paliouras, chair of the Duchenne Data Foundation: “The idea of structuring the event and the agenda based on questions from the community was a very good approach. The contribution of the participants in both the interactive sessions and the chat was great to see. Now that we are connected, we can push for change.”
Improving care in their country
Starting on Tuesday, Duchenne Patient Academy kicked off with sessions on what patient organizations can do to improve care in their country. Speakers sketched the bigger picture of the rare disease community by presenting outcomes of the H-CARE survey, and participants learned from success stories on implementing care from various rare disease and patient-led organizations.
Capacity & Policy
On Wednesday, experts were invited to share their experience on negotiation and professional communication. It became evident that in addition to ‘hard skills’ such as knowledge about clinical trials and updates on research, the community is interested in developing ‘soft skills’ such as emotional intelligence, communication skills, and conflict resolution.
Prof. Dr. Annemieke Aartsma Rus, Elizabeth Vroom and Eric Camino led the Thursday session on Therapy Development. Questions such as ‘Why don’t we have gene and stem cell therapy yet?’ and ‘What can patient organizations do to improve the patient voice in therapy development?’ were discussed in detail.
Data & Scientific Information
The Friday session was centered around Data & Scientific Information. Participants learnt about the importance of data from a research, clinical, and access perspective. Then, various speakers talked about how to connect, collect and reuse health data, and what role patient organizations can play in this. Deborah Robins closed the session with a presentation on how to find, follow and understand scientific information.
Project updates, Returning data, Company updates
The last session on Saturday of the Duchenne Patient Academy focused on project updates, returning individual clinical trial data, and company updates. Companies were invited to listen in and participate in the panel discussions on returning individual clinical trial data and pipeline discussions.
Duchenne Data Foundation thanks the companies supporting its educational programs for the global Duchenne Community: PTC Therapeutics, Sarepta, Roche and Italfarmaco.
What Duchenne Patient Academy participants say
“I plan on using the knowledge I have gained from the Academy to help with continuing my advocacy here in the United States to push for better care and inclusivity for the adult population living with Duchenne.”DJ Kimble, PPMD USA, United States
“Wonderfully organised event wherein we could finally communicate with world wide organisations like us. The interactive sessions gave us the broad aspect of how the problems such as lack of awareness are faced by most countries.”Anand Shah, PPMD India
“I appreciate the creative and very careful approach to the global DMD community – not only up-to-date information on the most problematic or advanced areas, but trust, warmth, respect to every member and organization.”Tatiana Gremiakova, CF Gordey, Russia
“I value the opportunity to attend the training and listen to stories from medical experts to organizations for patient advocacy. I am fortunate to be involved in the great journey to better everyday life for people living with Duchenne.”Vo Tan Phat, DMD Vietnam