2019 was a year where things have been given and taken from us. With this community letter, we hope to bring a birds eye view of this year, if it’s possible:
There are many events that happened this year that we can be proud of:
- In total, 14 new and existing patient organisations joined the global WDO community.
- The worldwide advocacy base has been strengthened with Duchenne Data Foundation’s Duchenne Patient Academy.
- The first Certified Duchenne Care Center in Europe is appointed to a hospital in the Czech Republic.
- We have little ones enrolling in gene therapy trials, something that we have only been dreaming of in the past years.
- We’ve been putting Duchenne adult care on the map and will continue improving this.
- A second drug Golodirsen has been approved by the FDA.
- We keep on finding new ways to improve quality of life, for example with nutrition and supplements, this year’s theme of World Duchenne Awareness Day.
With these highs, we’ve also hit some intense lows. Many brave boys and men passed away. Multiple trials have been discontinued due to safety or efficacy problems. Although gene therapy sounds promising, there are myriad challenges to overcome. Fragmentation and fake news divides the rare disease world, even in these modern times where we can have knowledge in the palm of our hands.
However, the year 2020 brings new opportunities for combination therapies, preclinical research and innovative trial design. New collaborations and projects have been initiated with other rare disease communities to share experiences. We continue our efforts to create an unified Duchenne registry, and implementing the FAIR Declaration to push open science.
Let’s create and remember beautiful memories. To the strongest families I know. May your days be merry, hearts be strong and minds determined to create a better future for all Duchenne families.