PatientsListening to Community Voices: XLH Café and Patient Conference Highlights

Kyowa Kirin is committed to raising awareness of X-linked hypophosphatemia (XLH) *1, a rare disease, and to creating opportunities for information sharing and dialogue to help patients and their families lead better lives. This article shares our ongoing initiatives and highlights events held in Japan and the United States in the fall of 2025, in conjunction with International XLH Awareness Day.

Kyowa Kirin has undertaken various XLH-related initiatives around the world. In Japan, we launched "Kurukotsu Hiroba" in 2020, a comprehensive website providing information on FGF23-related hypophosphatemic rickets and osteomalacia, including XLH. The site offers a wide range of content—from basic information on disease causes, symptoms, and diagnosis to medical subsidy systems and patient testimonials.

Currently, there is no patient association for XLH in Japan (as of April 2026). As a result, patients have limited opportunities to connect with one another, which has been identified as a challenge. In response to this need, we have been co-hosting a public lecture series called "XLH Café" since 2022 in partnership with ASrid, a nonprofit intermediary organization that supports collaboration among stakeholders involved in rare and intractable diseases.

The first XLH Café was held online, while the second (2023) and third (2024) events were held in hybrid format, combining online and in-person participation. At the second event, we organized a tea gathering after the lectures where participants could exchange information and ask questions to the speakers. One XLH patient shared: "I felt reassured knowing that others with the same disease were gathered together." Participation grew from 30 families at the first event to 45 in 2023 and 60 in 2024.

The XLH Café held on October 19, 2025, expanded further in scale, with on-site venues in Tokyo, Osaka, Nagoya, Okayama, and Fukuoka, as well as online participation. A total of 64 families attended (approximately 100 attendees). During the first two hours, a video featuring patient stories was shown, followed by lectures by medical specialists on three themes: “What Everyone Should Know About XLH,” “Treatment and Complications to Be Aware Of,” and “The Importance of Dental Care.” This provided an opportunity to share a broad range of knowledge with patients and their families. For the first time, a patient living with XLH gave a video message, which participants said helped them feel the presence of others facing the same disease. After the lectures, one-hour tea gatherings (conducted by ASrid) were held at all venues, including online, serving as a space for building connections.

In the United States, The XLH Network held its Annual XLH Day Patient Conference in Las Vegas in October 2025. This marked the third consecutive year that Kyowa Kirin has sponsored the event, reaffirming its commitment to supporting XLH education and opportunities for community connection. The conference brought together a diverse group of participants, including patients and their families, caregivers, healthcare professionals, and Kyowa Kirin employees. Of the more than 100 attendees, 80 were first-time participants, including two families who were meeting other XLH patients for the very first time.

The conference featured a keynote address by Neena Nizar, a patient advocate living with a rare bone disease. Nizar founded an advocacy organization dedicated to supporting families affected by the same condition and advancing research toward new treatments. Drawing on her personal journey, she spoke about the critical role of self-advocacy for people living with rare diseases—encouraging attendees to understand their hopes, needs, and rights, to voice them confidently, and to seek the support necessary to navigate their healthcare journeys. In addition to the keynote, the conference offered sessions covering a wide range of practical and community-centered topics, including:

During the conference, Kyowa Kirin had a table to connect with attendees and share updates on past and ongoing initiatives. This included progress on a community guidebook, currently in development, where attendees were invited to provide feedback on proposed topics and content. We also conducted video interviews with community members and displayed results from the XLH Community Impact Survey, conducted jointly with The XLH Network.
These exchanges provided valuable insights and underscored the importance of continued collaboration with the XLH community.

Abdul Mullick, CEO of Kyowa Kirin, who traveled from Japan, shared: "Experiencing firsthand the strength and spirit of the XLH community allowed us to listen, learn, and truly connect — to see the smiles, hear the stories, and feel the impact of what we do. Every interaction reminded us that behind every data point is a person, a life we have the privilege to touch. Their courage inspires us to keep advancing care and creating more smiles for those living with rare diseases."

The initiatives supporting XLH patients in Japan and the United States provided an opportunity not only for patients and their families, but also for healthcare professionals and Kyowa Kirin employees, to deepen connections and strengthen the shared desire to "move forward together." Tomoko Nakayama, who leads this patient advocacy activity at Kyowa Kirin in Japan, said: " XLH Café is a collaborative event, co-created from the planning stage by patients and families, supporters, healthcare professionals, and pharmaceutical companies. By serving as a starting point for connections among patients, XLH Café aims to create new value and hope for each participant and will continue as an ongoing initiative."

Liz Butcher, Associate Director, Patient Advocacy, Engagement, and Policy, Rare Disease, Kyowa Kirin, U.S., shared with a smile:

"Our partnership with the XLH patient community is at the heart of everything we do. It's incredibly rewarding to see the community come together and share stories about the meaningful value Kyowa Kirin brings in the development of so many collaborative initiatives."

Kyowa Kirin will continue to listen to the voices of patients with XLH and other rare diseases, and walk together with them.

The activities mentioned in the article were conducted in each region under the relevant laws and regulations of each country.