Champions of Hope: The Heartfelt World of Patient Advocacy in Rare Diseases

In the vast tapestry of healthcare, certain threads weave a tale of strength, resilience, and unwavering hope. This narrative belongs to the patient advocates who champion the cause of those navigating the challenging terrain of rare diseases and pediatric cancer.

Patient advocacy is more than a buzzword; it's a beacon of support, a lifeline for families facing the unknown.

Round table about Achondroplasia, Belgrade, December 2023

Can you explain what "Patient Advocacy" means?

Patient advocacy is the unwavering commitment to empower and support individuals and families facing the challenges of rare diseases and pediatric cancer. It involves raising awareness, providing information, and amplifying the voices of those who often find themselves in the shadows of the medical landscape.

Activities that Define Advocacy:

Raising Awareness: Advocates work tirelessly to shine a light on rare diseases and pediatric cancer, dispelling myths and fostering understanding within communities and the medical field.

Educating the Public: Patient advocates strive to bridge the knowledge gap, offering valuable information about specific conditions, treatment options, and the emotional toll these diseases take on families.

Navigating Healthcare Systems: Advocates serve as guides through the labyrinth of healthcare, helping families access resources, understand treatment plans, and overcome bureaucratic hurdles.

Fostering Community: Creating a network of support is crucial. Advocates bring together families facing similar challenges, fostering a sense of community that is both empowering and comforting.

Influencing Policy: Advocates often work with policymakers to shape healthcare policies, ensuring that the unique needs of those affected by rare diseases and pediatric cancer are considered.

Conference about neurotransmitter diseases, Belgrade, October 2022

People in Patient Advocacy who I admire

Besides persons I now can call my friends, and who gave their time and resources to improve the quality of life of rare disease patients in Serbia, there are many more whom I admire and follow in their work.

• Pat Furlong (Parent Project Muscular Dystrophy): A tireless advocate for those with Duchenne muscular dystrophy, Pat Furlong founded the Parent Project Muscular Dystrophy, driving research and support for families.
• Nancy Goodman (Kids v Cancer): Nancy Goodman's advocacy led to the passing of the Creating Hope Act, encouraging pharmaceutical companies to develop treatments for pediatric cancers. This was very helpful in the moments of despair and sadness dealing with Viktor's therapy.
• Kevin Alexander (Host of "Never Give Up": A Rare Disease Podcast): Kevin is a PKU patient, but that didn't stop him from pursuing a career and sharing his story with the world. He is doing a lot for the PKU community globally, and I have learned a lot from him.

The advocacy in the media is a must!

You can help us too...

Patient advocacy is a powerful force that illuminates the darkest corners of rare diseases and pediatric cancer.
The advocates mentioned above, along with countless others, exemplify the spirit of resilience, compassion, and optimism that defines this field.
If you have questions or want to share your experiences, I'm here to listen.

Let's build a community of support and hope. Feel free to reach out, and together, let's continue to be champions of hope in the face of adversity.