Rare disease group finds advocacy and looks for answers
- Date: April 26, 2018
- By: .
SIOUX FALLS, S.D. –- A dedicated Board of Directors for an international nonprofit organization representing those who have Alagille Syndrome, a are genetic condition that can affect the liver and other organs, recently met with the Sanford Health researcher hoping to cure their disease.
The Alagille Syndrome Alliance, which began in 1993 as a newsletter to two families, has grown to a nonprofit that serves families around the world looking for advice, advocacy and answers for their genetic conditions. The international board includes members from the U.K., Canada and India, as well as the United States.
In 2017, the ALGSA board decided to migrate its data to Coordination of Rare Disease at Sanford (CoRDS), a free database which began in 2010 and now serves nearly 5,000 people representing more than 770 rare diseases. The free registry offered them an opportunity to create disease-specific questionnaires and connect to Sanford Research laboratories and specialists.
“CoRDS is so proactive about data collection and communication,” said Cindy Luxhoj, founder and executive director of the Alagille Syndrome Alliance. “CoRDS also provides our families and those affected by ALGS an opportunity to meaningfully contribute to research and move ALGS science forward toward a cure.”
The group took it a step further earlier this year, when several members traveled to South Dakota to meet with Kameswaran Surrendran, Ph.D., an associate scientist with Sanford Health who is doing research on Alagille Syndrome.
“Meeting with him was fantastic,” Luxhoj said. “He had all these questions for us, and we had so many for him.”
Anna Laurent, who has Alagille Syndrome, agreed. She met with Surrendran and also connected with others from ALGSA at a conference in Sioux Falls in 2018.
“In a world where your disease is so rare that you are 1 in 70,000, it was such a big moment to know that I was not alone,” Laurent said of her many experiences connecting with other families over the years. “When you meet these young warriors who are singing to ‘Frozen,’ singing Disney songs, and their parents are just starting on this journey, that’s the reason we need research.”
Researching rare diseases and bringing patients, scientists and physicians together is an integral part of what Sanford Health does, said David Pearce, Ph.D., executive vice president of research and innovation at Sanford.
“I see these families, and I see how they are searching for answers, and how their lives have been affected,” Pearce said. “We know we can make a difference, and every day we are asking questions and working with different groups to find answers.”
Sanford Health physicians and leaders will be among the international experts presenting on rare diseases and other topics at the Fourth International Vatican Conference in Rome, Italy, on April 26 – 28.
The conference, “Unite to Cure: “The Fourth International Vatican Conference – How Science, Technology and 21st Century Medicine Will Impact Culture and Society,” brings together leaders in health care, science and research from around the world as part of the Cura Foundation conference, which is held every other year in Rome. This is the second time Sanford Health has presented at the invite-only event. Robin Smith, M.D., president of the foundation, also serves on the Sanford International Board.
“What we are doing today could actually impact someone’s life,” Surrendran said. “There is still a long way to go, but every day we are doing something meaningful.”
To learn more about Unite To Cure: The Fourth International Vatican Conference, please visit: vaticanconference2018.com. Or, you can follow the event on Twitter @CuraFdn and on Facebook at facebook.com/TheCuraFoundation, and join the conversation with #UnitetoCure.
About Sanford Health
Sanford Health is one of the largest health care systems in the nation, with 44 hospitals and nearly 300 clinics in nine states and nine countries. Headquartered in Sioux Falls, South Dakota, and serving the Upper Midwest, with nearly 1,400 physicians, Sanford Health is dedicated to several initiatives, including global clinics, genomic medicine and specialized centers researching cures for type 1 diabetes, breast cancer and other diseases. Sanford Health has 28,000 employees, making it the largest employer in the Dakotas. Nearly $1 billion in gifts from philanthropist Denny Sanford over the past decade have transformed how Sanford Health can improve the human condition. For information, visit sanfordhealth.org.
About the Cura Foundation
The Cura Foundation leads a major global health movement, with the passionate purpose to improve human health. Cura unites public and private sectors, partnering with doctors, patients, business leaders, philanthropists and thought leaders to collaborate and create breakthroughs around the world. The foundation drives change by raising awareness of scientific advancements in genomics, emerging technologies and big data to usher in the future of medicine. Cura helps people live longer, better lives free from suffering. The Cura Foundation is a nonsectarian, nonpartisan, public and tax-exempt organization under Section 501(c)(3) of the Internal Revenue Code. For more information, please visit: https://thecurafoundation.org
About ALGSA
The ALGSA is an international nonprofit based in Oregon. We mobilize resources, facilitate connections, promote unity, and advocate for a cure to inspire, empower, and enrich the lives of people affected by Alagille Syndrome (ALGS). We are a driver of ALGS advocacy, awareness, patient support, and research. We represent a vibrant community and provide a vital link among families, as well as offer them the resources they need to help their children live longer and healthier lives. Our vision is that all people affected by ALGS thrive in a close-knit community full of loving support, easily accessible resources, and life affirming hope. For more information, visit the ALGSA website at www.alagille.org, email to alagille@alagille.org.
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