HypoPARAthyroidism Association Launches the First-of-its-Kind Patient-Powered Data Platform to Advance Patient Care

Tuesday, June 21, 2022

HypoPARAthyroidism Association, Inc., an organization dedicated to improving the lives of people impacted by hypoparathyroidism (HypoPARA) through education, support, research, and advocacy, today announced the launch of the first-of-its-kind Patient-Powered Data Platform. The platform will aggregate data voluntarily entered by patients about their disease, medical history and quality of life, with the long-term goal of building a patient-driven data repository that can help healthcare providers, researchers and clinicians make informed decisions about the treatments for people living with HypoPARA. This launch is a critical first step to broadening the HypoPARA research journey and an important resource for patients and their caregivers to advance care for the HypoPARA communities.

HypoPARA is a disease that causes lower than normal levels of calcium in the blood due to insufficient levels of parathyroid hormone (PTH), in turn affecting the health of bones, kidneys, cardiovascular system, gastrointestinal system, and brain. This condition may result from neck surgery. It can also be inherited or associated with other disorders. HypoPARA affects an estimated 100,000 people in the United States, including males and females in equal numbers. Many patients living with HypoPARA feel its effects on their health and quality of life every day of the year and need new therapies that treat the underlying disease.

“Few clinicians and healthcare providers have direct experience treating patients living with this rare disease, and so few understand its impact,” said Dr. Dolores Shoback, Medical Advisor at HypoPARAthyroidism Association. “The Patient-Powered Data Platform is a new tool that we are certain will become an essential community-driven resource for advancing collective understanding of HypoPARA, supporting the safe and rapid development of new therapies and better treatment paradigms to improve the health and quality of life for patients. I hope those within the medical and healthcare communities who have contact with these patients will encourage them to learn more about the Patient-Powered Data Platform and consider participating in it.”

The Patient-Powered Data Platform is being developed in partnership with RARE-X, a nonprofit organization created by leaders in the fields of patient advocacy, medical research, biopharma, technology, and other patient support organizations from around the world. RARE-X is leveraging existing technology powered by the Broad Institute of MIT and Harvard and other technology partners, which will support patients (in data collection, structuring and responsible sharing), clinicians (identifying HypoPARA specialists/researchers; accelerating diagnosis and improving and tracking health outcomes), researchers and biopharma (with the data they need to identify, develop, and track the impact of breakthrough treatments and cures). As RARE-X is a nonprofit organization, there is no cost to members of the HypoPARA community who wish to participate in advancing the understanding and treatment of this serious medical condition.

New therapies that safely and effectively address the underlying biology of HypoPARA and can improve patients’ quality of life are urgently needed. The Data Platform is designed to inform researchers’ understanding of how the symptoms of HypoPARA, or response to therapy, change over time. This will enable better clinical trial design and data collection strategies, support the use of natural history data instead of placebo in clinical trials, and facilitate patients’ participation in clinical trials. The longer-term vision for this platform is to reduce the time it takes to study new potential medicines and speed up the availability of new treatments to patients.

“Understanding the signs, symptoms, diagnostic criteria, causes, and treatment paradigms for HypoPARA is critical for providing patients with optimum care, and systematically collecting such data is critical for enabling the innovation necessary to develop new treatments,” said Deb Murphy, Director of Research and Studies, and former President at HypoPARAthyroidism Association. “The Patient-Powered Data Platform will also make it easier to identify endocrinologists and other specialists who may have expertise in treating HypoPARA. Ready access to such information is important for helping to ensure that known or suspected HypoPARA cases are diagnosed accurately and treated effectively on time.”

To learn more about the Patient-Powered Data Platform, please visit https://hypoparathyroidism.rare-x.org

About HypoPARA
Hypoparathyroidism is a disorder that causes lower than normal levels of calcium in the blood due to insufficient levels of parathyroid hormone (PTH). This condition may result from neck surgery. It can also be inherited and/or associated with other disorders. Hypoparathyroidism affects an estimated 100,000 people in the United States, including males and females in equal numbers.

PTH plays an essential role in regulating serum calcium and phosphorous levels, and patients with HypoPARA have reduced serum calcium (known as hypocalcemia), excess serum phosphate (hyperphosphatemia), and excess calcium in urine (hypercalciuria). Given the critical function of calcium and phosphate in various biologic pathways, alterations in the levels of these minerals negatively affect diverse organ systems and lead to kidney stones, chronic kidney disease, muscle cramps, cognitive dysfunction, seizures, arrhythmias, wheezing, and cataracts. As a result of these symptoms, patients living with HypoPARA often have diminished quality of life.

Conventional therapy for HypoPARA aims to raise serum calcium into the lower half or just below the normal reference range, which is intended to alleviate symptoms of hypocalcemia, avoid hypercalciuria, and maintain normal serum phosphate levels. This is typically achieved through the lifelong use of calcium supplements, such as calcium citrate or calcium carbonate. However, this therapy only addresses symptoms in some patients. HypoPARA is associated with calcification of soft tissue and organ systems and can specifically affect the kidneys, but the heart, gastrointestinal, and nervous system can sometimes be involved.

About HypoPARAthyroidism Association, Inc.
HypoPARAthyroidism Association, Inc., is dedicated to improving the lives of people impacted by hypoparathyroidism (HypoPARA) through education, support, research, and advocacy. We strive to be a reliable, dedicated, accessible, inclusive, empowering resource while being compassionate and professional.

Media Contact
Glenn Silver
Glenn.Silver@finnpartners.com
(646) 871-8485

Latest Issue
Get instant
access to our latest e-book
Sanofi Elsevier - Bring ClinicalKey to your institution Medical Manufacturing Asia 2022 Medical Fair Asia 2022