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David Fajgenbaum David Fajgenbaum Every Cure

If being a rare disease patient has taught me anything, it’s that you need to be your own advocate.

There’s a common desire among us when facing health challenges to find a doctor who can provide all the answers, allowing us to simply follow their guidance and not worry about the intricacies of our conditions.

However, my own journey has taught me the value of being an active participant in my treatment plan. Had I adopted a passive approach, accepting the first set of answers without question, my story might have ended differently.

This isn’t about placing undue pressure on ourselves to become medical experts or single-handedly revolutionize treatment options.

Rather, it’s about recognizing the power we hold when we engage actively with our care, seeking out specialized centers, connecting with relevant foundations, and exploring all avenues for the best possible care.

I would love to learn in the comments how have you taken an active role in your healthcare journey!

Updated 11 hours ago
Canary Advisors Canary Advisors Canary Advisors

Canary Advisors
Patrick O'Connor Patrick O’Connor Uplifting Athletes

Check out the Minnesota Twins’ Target Field lit up for Rare Disease Day!

Twins Field

Paula Penney Admin Paula Penney Worldwide

My nephew Steffen was born with a rare genetic disorder -CHARGE Syndrome- 22 years ago. Although he is deaf, blind, and has multiple other issues, he is not medically fragile. Steffen is nonverbal and has Autistic tendencies. Nothing holds Steffen back. Whatever the family does, so does Steffen. Whether it is whitewater rafting, skiing downhill, skating, or a dance video party, Steffen is right there. His laugh and joking nature are a joy to be around. Thanks to great health care providers and interveners/caregivers, Steffens life is full and brings joy and love to all those that know him. His parents are members of the CHARGE Syndrome foundation and provide support to new parents who receive the rare diagnosis of CHARGE. It means everything to talk to someone who has been there. Today is special day for our family. Together we can raise awareness and influence change.

Amy Getman Amy Getman Worldwide Clinical Trials

On this Rare Disease Day, I pause and give gratitude to all those in our community who help move the needle in researching and fighting Rare Diseases. A very close friend of mine was diagnosed with NMO (Neuromyelitis optica) several years ago and fortunately has found a medicine that has kept her in remission from relapses. She goes every two weeks for an infusion and will continue this for the rest of her life. It amazes me the resilience and positive attitude she carries with her in the face of this terrible rare disease. I love working for a passionate organization that helps companies execute Rare Disease trials in hopes of finding new treatments for these patients!

Deborah Requesens Deborah Requesens Orphan Disease Center

⭐ Today is #RareDiseaseDay! Join us as we #leap4rare to show our support for the 300 million people worldwide living with rare diseases. The Orphan Disease Center is dedicated to advancing research and providing support all year round. Let’s unite in solidarity and make a difference together! #raredisease #supportresearch
Trinity Life Sciences Trinity Life Sciences Trinity Life Sciences

Read our latest blog post to learn about the cell and gene therapy trends Trinity Life Sciences is watching for 2024!
Amy Raymond Amy Raymond Worldwide Clinical Trials

This Rare Disease Day, I am thinking about how phenomenal it is to see how rare oncology patients have benefitted from the 6 CAR T treatments now available! It is exciting to watch how continued advancements have turned CAR T into a new development paradigm for autoimmune diseases – looking forward to seeing these additional patient communities benefit from cell therapy!

Rob Long Rob Long Uplifting Athletes

In celebration of Rare Disease Day, I am excited to share our most recent draft class from the 2024 Young Investigator Draft! This year we highlighted the significant potential of research in this space when powered by the platform of sports—especially as we surpassed our $1 million in total funding granted. You can watch a recap of the event at the below link!

John Morris John Morris International WAGR Syndrome Association

When my daughter was diagnosed with WAGR syndrome, none of the doctors that were treating my daughter knew anything about WAGR. Since that time, I’ve dedicated my time and energy to advocate for my daughter, spreading awareness of WAGR throughout my local and the international communities.
Joy Clark Admin Joy Clark Worldwide Clinical Trials

Rare diseases affect hundreds of millions of people globally, presenting a massive challenge to health equity. Our team is focused on changing that sad reality for patients across many therapeutic areas. From rare cancers such as the one that took my sweet cousin Ryan last year to rare cardiovascular diseases, I’m proud of how hard our teams work every single day to improve the standard of care for rare disease patients. Their passionate and purposeful commitment to saving and improving lives is not just a job; it’s creating meaningful change for our loved ones, their caregivers, and for humanity as a whole.

Rare Disease
Rare Disease


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