Liam McCarthy
Members of Rare Diseases International’s Youth Leadership Programme
Pablo Ramirez Uribe
Members of Rare Diseases International’s Youth Leadership Programme
Youth advocates with lived experience of rare diseases are mobilising globally to drive positive change.
Of the almost 7,000 rare diseases, 70% appear during childhood, and only 5% have a recognised treatment.
Reality of rare diseases for young people
As young people living with congenital rare conditions, we understand its challenges: the fear of an early death, lack of medical knowledge, questioning from physicians and financial strains on our families. Still, we’ve been fortunate to have access to the treatments we needed to survive.
Millions of people living with rare diseases struggle to access diagnosis, treatment and care. We want to use our voices, perspectives and strengths to reduce these inequities and improve the lives of people living with a rare disease globally.
As young rare disease advocates, we know that the
youth can change the world if given the platform.
Mobilising global action
The rare diseases landscape is at a critical moment: last May, the World Health Assembly (WHA) passed a landmark resolution recognising rare diseases as a global health priority and calling for the development of a 10-year Global Action Plan for Rare Diseases (GAPRD) to be developed over the next two years.
As participants in Rare Diseases International’s Youth Leadership Programme, we travelled to Geneva to witness this historic event alongside 11 other youth advocates from around the world.
Now we stand ready to participate in the next steps for ensuring that the Resolution has a real impact in areas where people living with a rare disease need it most. Youth voices are critical. As the GAPRD will impact the rare disease landscape over the next decades, it must respond to our concerns and needs, and we must actively participate in shaping it. As young rare disease advocates, we know that the youth can change the world if given the platform. We’re ready to seize every platform — whether by sharing our stories on social media or speaking at regional and global events — to raise our voices alongside more established leaders. When young leaders are given space to be heard, we can drive meaningful, global change to shape our future.
[1] The Lancet Global Health. (2024). The landscape for rare diseases in 2024.
