Rare disease means sharing both challenges and advances

One condition can change many lives. That’s something we observe over and over in rare kidney disease (RKD). One person gets a diagnosis, and an entire family –?or even community –?can feel the impact. In that way, facing a rare disease is often a shared experience.

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Living with a rare kidney disease can include many difficult shared experiences, so when there is good news to share, we deserve to celebrate that together as well. As we announce an approval milestone for a treatment that is already making an impact in IgA nephropathy, I’m humbled to reflect on the small but mighty village of dedicated people who have helped make this possible.

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I’m grateful to the community united by experiences of RKD. Whole families lovingly shape their lives and schedules to accommodate new pressures and demands as they grapple with the emotional toll of knowing a person they love is struggling. They also feel and express hope — matched with a sense of urgency for more options. In my very first month at Travere, I had a lifechanging encounter with a mother of a young man with a rare kidney disease. I was touched by how much she was doing to advocate for her son. I was even more moved when she looked me in the eye and asked, “What are YOU going to do for my child?”

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Her challenge motivated me, and in the years since, I’ve continued to find inspiration in the energy and optimism of the advocacy community. Progress has historically been slow in RKD, and while patients and families waited for treatments, organizations like NephCure and the IgA Nephropathy Foundation mobilized, not hesitating to create networks for information sharing, support, and understanding. Advocacy organizations spread the news about ongoing innovation, bringing awareness and encouragement to this field, and challenging us to aim higher. They are also generous with their time, helping organizations researching these conditions by sharing insights that inform research as well as offering personal stories that put weight behind our work. Their work helps to keep hope alive, and I’m grateful for it.

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In addition to empowered patients and activated advocacy organizations, I also want to thank the dedicated nephrologists and healthcare providers who stepped up, expecting more for their patients, recognizing people living with IgA nephropathy deserve treatment options. There are so many who have dedicated their careers to advancing this field of medicine - and their achievements are many. We hear stories of providers’ and patients’ resilience every day as they work together to find the best possible solution to slow disease progression.

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And I want to thank the investigators whose efforts help us build to moments like this. Just as the families of patients come together, there is a palpable sense of shared mission within the RKD research space.

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Finally, to our team at Travere – thank you for your passionate work and thoughtful collaboration that brought us to this point, and that will continue propelling us forward. Today’s approval is going to impact so many lives and make other progress possible. Thank you for paving a way forward where there wasn’t one before.

#IgAN #InRareForLife