2022 Wrap-up: Activities of World Duchenne Organization

Yearly wrap-ups with a series of predictions of what 2023 will bring us are common at this time of year. However, looking back at the past two years we can all agree that predicting and horizon scanning are pretty much impossible. Especially in the field of healthcare and rare diseases. We are closing a year where lots of good and sad things have happened. Elizabeth Vroom looks back on 2022 for the World Duchenne Organization, listing all activities of the World Duchenne Organization, and carefully draws a rough outline for 2023.

Our underlying motivation for being active in this field is well-defined. We want to make the lives of people living with Duchenne and Becker muscular dystrophy easier, better and happier. Day after day, it is made clear that there is a lot to gain in terms of quality of life, access to care and therapies, and participation in society. In order to successfully advocate for this, you need a well-educated and strongly connected community. This is at the core of what we do at World Duchenne Organization.

As an organization, we share the information, connect the community, and develop educational programs. Behind all our activities, there is an ambition to find new and better ways to do things. Each new insight leads us one step in the direction of better understanding how to treat the disease that connects us all. We are immensely grateful for all our member organizations who are part of working towards this goal.

World Duchenne Organization in 2022

In 2022, the World Duchenne Organization activities included to:

• Organize the 2022 WDO Members Meeting
• Activate the DMD Emergency Program for families affected by the war in Ukraine
• Organize the Duchenne Care Conference 2022 on Adult Duchenne Care
• Award Prof Muntoni the WDO Leadership Award celebrating exemplary leadership
• Organize a successful World Duchenne Awareness Day event on Women and Duchenne
• Host the 3rd International Muscle-Bone Interaction in Duchenne Muscular Dystrophy Symposium in collaboration with ICCBH
• Organize a meeting on FAIR Data ‘Visiting’ for Duchenne and other Rare Diseases
• Announce the Accredited Duchenne Center Program for worldwide accreditation for Duchenne centers
• Welcome new WDO Member and WDO Aspiring Member organizations
• Develop and host Duchenne Patient Academy 2022 in collaboration with Duchenne Data Foundation
• Provided the environment for discussions between pharmaceutical companies with patient experts with the Duchenne CAB
• Actively be involved as partner in European funded projects such as BEAMER, BIND, EURO-NMD Registry Hub and Trials@Home
• Share Prof Aartsma-Rus’ paper-a-days, where she reads and comments on scientific publications

I would like to share a quote by Søren Kierkegaard from 1843, who was a Danish philosopher. “Life can only be understood backwards, but it must be lived forwards”. There are two parts in this quote that appeal to me. First and foremost, only when you see the greater picture, you can see patterns. This is why it is important to be together so we can share experiences and stay connected. Secondly, we should live in the present and think in opportunities. Cherish the moments. This is sometimes more difficult than others. But it is an important insight if you want to break the cycle of doubt and uncertainty.

Looking forward to 2023

In the next year, we aim to focus on the following things:

• Discuss topics WDO should focus on with the WDO Members Meeting
• Provide an update on care, including females with dystrophinopathies in the Duchenne Care Conference
• We celebrate a milestone of the 10th anniversary of World Duchenne Awareness Day
• Continue with our efforts to advocate for Newborn Screening in Duchenne
• Help patient organizations to connect with, or create, their own FAIR Registry
• Continue to empower patient organizations, families and people living with dystrophinopathies to advocate for their right to live life to the fullest
• In the field of drug development, we will continue with the Duchenne CAB and initiate multi-stakeholder meetings involved in drug development

Last but not least, if you have a view about what World Duchenne Organization should look at, what we should say or other questions that we should be asking ourselves, please get in touch. Otherwise, keep an eye out for the activities and news.

To summarize, we create a global and tighter network of devoted patient representatives, including advocacy groups who are not official organizations through the Aspiring Membership. We continue to devote ourselves on raising awareness for dystrophinopathies. 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.

 

Elizabeth Vroom, Chair
on behalf of the World Duchenne Organization