American Kidney Fund Launches New Education Initiative for People Living with Cystinosis and Their Families


Digital and print resources focus on rare disease that causes kidney dysfunction in infants and children

ROCKVILLE, Md. (Feb. 24, 2020) – The American Kidney Fund (AKF) today announced a new educational initiative to support people living with cystinosis, a rare, multisystem genetic disease that leads to kidney failure in the majority of people living with the disease.

The AKF initiative, supported by a grant from Horizon Therapeutics plc, will provide patients and their caregivers with expanded content on AKF’s website and digital communications, including information about preparing pre- and post-transplant. A patient and caregiver information toolkit that empowers patients to adhere to treatment, prepare for transplant and life post-transplant, and engage them in staying as healthy as possible is being distributed to specialists. The toolkit was developed in cooperation with the Cystinosis Research Network and has sections with information specifically for parents and teens. Both can be downloaded from AKF’s website.

The most common and severe form of cystinosis causes kidney failure in children by about age 10, and most children with cystinosis will eventually need a kidney transplant. The majority will need two or more transplants in their lifetime, because a transplant does not cure cystinosis. AKF’s educational materials will help families understand the process of getting kidney transplants for their children. It offers practical information on helping their children cope with the lifestyle, setting up a caregiving team, what to tell the school when the child needs to take time off, and transitioning responsibility to the child as they reach adulthood.

“Cystinosis occurs in only about 1 in 100,000-200,000 children, so it is not widely known or understood,” said LaVarne A. Burton, president and CEO of the American Kidney Fund. “We recognize parents may be overwhelmed when their child is diagnosed with this rare disease, and we are designing our education resources to help them navigate their most pressing questions, including how to help their child prepare for the eventuality of a kidney transplant. We are grateful to Horizon for its support in this important effort.”

“Horizon is passionate about positively impacting the lives of people living with rare diseases, like cystinosis, and proud to support the educational programs led by the American Kidney Fund,” said B.J. Viau, director, patient advocacy, Horizon. “Families impacted by cystinosis are the true experts of the condition and we hope these materials, created in partnership with the cystinosis community, can be added to the toolbox of resources.”

About the American Kidney Fund

As the nation’s leading nonprofit working on behalf of the 30 million Americans with kidney disease, the American Kidney Fund is dedicated to ensuring that every kidney patient has access to health care, and that every person at risk for kidney disease is empowered to prevent it. AKF provides a complete spectrum of programs and services: prevention outreach, top-rated health educational resources, and direct financial assistance enabling 1 in 5 U.S. dialysis patients to access lifesaving medical care, including dialysis and transplantation. AKF holds the highest ratings from the nation’s charity watchdog groups, including Charity Navigator, which includes AKF on its “top 10” list of nonprofits with the longest track records of outstanding stewardship of the donated dollar, and GuideStar, which has awarded AKF its Platinum Seal of Transparency.

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About Horizon

Horizon is focused on researching, developing and commercializing medicines that address critical needs for people impacted by rare and rheumatic diseases. Our pipeline is purposeful: we apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, please visit, follow us @HorizonNews on Twitter, like us on Facebook or explore career opportunities on LinkedIn.