2021 Wrap-up: Reflections, Activities, Moving Forward
The year 2022 is greeting us, bursting with new opportunities. Before waving goodbye to 2021, Elizabeth Vroom is reflecting back on the year. This wrap-up contains an overview of the projects and activities of the World Duchenne Organization in 2021.
We are looking back on a year counting our successes and losses. Too many brave young men have passed away too early. It is a constant reminder of how much work is still ahead of us. The need to share timely and truthful information with all people and organizations is clear, sometimes painfully clear.
The World Duchenne Organization will continue to promote Duchenne and Becker MD research, data (re)use, and support the development of tools to improve how clinical trials are conducted. Together we will work to ensure all people living with the disease have access to the best care, and new drugs and treatments. A relatively young field is currently being explored by involving people living with Duchenne and Becker MD in activities and decision-making.
In 2021, the World Duchenne Organization has been successfully focusing on various topics and activities, such as:
- Welcoming 10 organizations to join the global community
- Published several articles on the WDO website, e.g. whether the COVID-19 vaccine affects the possibility to have gene therapy or the dangers of unproven stem cell therapy
- Co-authored various papers, e.g. the Whitepaper on Returning Individual Clinical Trial Data Back to Participants
- WDO Webinars on COVID-19 and Duchenne
- Adding new languages to the DMD-Care videos
- Presented at various international conferences on rare diseases, pediatric drug development, data, and neuromuscular conditions.
- February: WDO Member Meeting (virtual)
- March: FAIR Data for Duchenne meeting
- June: Duchenne Care Conference 2021
- July: Meeting on Wearables on the use and development of wearables, and other digital outcomes for Duchenne
- September: World Duchenne Awareness Day 2021
- Map and support various activities across the globe
- Livestream Adult Life & Duchenne
- Share the Consensus Guideline on Adult Duchenne Care
- November: Co-organize Duchenne Patient Academy 2021
- Advocate for Newborn Screening in Duchenne
- Optimize-DMD to harmonize and optimize the DMD development pathway from research to access
- Groups Engagement to create solutions to involve all groups from all countries
- Completed an Emergency Program to aid Lebanon after the Beirut Blast
- Developed a program to Democratize Access to Information
- Involving the global community in EU projects to:
- Improving patient adherence to prescribed treatments through BEAMER;
- Assess and address learning and behavior issues in DMD/BMD with BIND;
- Develop a patient-centered hub to improve care for (undiagnosed) neuromuscular diseases through Euro-NMD Registry Hub;
- Design a community for patient-led research in rare diseases with Share4Rare;
- Create an External Stakeholder Panel to discuss decentralized clinical trials in [email protected]
We understand that 2021 was a difficult year for us all, for some groups even more. The World Duchenne Organization is grateful for her members. An ever-growing community who works tirelessly to improve the lives of so many Duchenne and Becker families around the world. Thank you for your time, input and energy. In the new year, we can’t wait to present you the program of 2022, that is co-created with you.
For scientists and clinicians
In addition to our organizations, we count our blessings by being able to tap into the vast amount of knowledge. We are fortunate to collaborate with the best scientists and clinicians around the globe, who work on sharing the newest updates in an understandable format.
In the new year, we will continue to grow and devote ourselves on raising awareness for DMD/BMD. We are increasing WDO’s presence and visibility, and above all work to finding a cure and viable treatments, to promoting good standards of care, and to inform parents and people living with the disease around the globe.
In conclusion, we are ready for 2022, full of opportunities to take new steps and create a brighter future for people living with Duchenne and Becker muscular dystrophy!
Elizabeth Vroom, Chair
on behalf of the World Duchenne Organization
Photo by Mike Labrum on Unsplash