Hope and Determination on Rare Disease Day 2024

Hope and Determination on Rare Disease Day 2024

Today, 29th February 2024, marks Rare Disease Day. At Action Duchenne, we join with the global rare disease community to shine a light on the 300 million people across the world living with rare diseases and their families.

Duchenne muscular dystrophy is one of the 70% of genetic rare diseases that begin in childhood. Like many of these conditions, Duchenne currently has no cure and the treatment options remain limited. For those living with Duchenne across the world, many face inequalities in access to social opportunities and healthcare as well as isolation and lack of support. 

Our work continues to focus on supporting families from the moment of diagnosis, offering much-needed psychological support, connection with other families and empowering science education. Our transition programmes provide opportunities for young people living with Duchenne to fulfil their potential and to challenge society’s view of the capabilities of those living with disability. We continue to fund research to drive forward the hope for improved treatments, to fight for access to those treatments and to be the voice for the Duchenne community. Today we stand with the global rare disease community with real hope for the future and with determination for it to be realised. 

On Rare Disease Day 2024, I would like to leave you with some inspirational words from a young man living with Duchenne which not only give us all hope for the future but reminds us what we are fighting for. 

“I want to tell my 10 year old self and all those boys living with Duchenne that anything is possible if you want it, and that you can live your dream.” Sanjeev Mann 

As always, the team here at Action Duchenne are here for you and your family. We will continue to be your voice in the rare disease community and to work tirelessly towards a future where lives are no longer limited by Duchenne muscular dystrophy.