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Note: This story was updated July 13, 2022, to correct the name of Rare-X’s CEO Charlene Son Rigby. Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world.
The HypoPARAthyroidism Association has launched the first-of-its-kind Patient-Powered Data Platform, a repository to help understand the disease journey and develop better treatments for people with hypoparathyroidism. Interested participants — patients, caregivers, or those who have lost a loved one to the rare disease — can enter patient…
Children successfully treated for a cancer who undergo thyroid surgery for a secondary thyroid disease have a similar risk of surgical complications, such as permanent hypoparathyroidism, as children without a previous cancer, a single-center study in the U.S. shows. These findings add to an ongoing discussion of whether cancer…
The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…
Daily treatment with AZP-3601, Amolyt Pharma’s investigational therapy for hypoparathyroidism, was well tolerated and allowed patients to discontinue standard supplement treatment, early data from a Phase 2a trial show. The treatment also rapidly normalized 24-hour urine calcium levels and improved bone health. The findings, from a first group of…
Late recovery — longer than six months — from post-surgery hypoparathyroidism is not uncommon, and having levels of parathyroid hormone (PTH) within the normal range may indicate whether patients are more likely to recover, a study found. “PTH is a useful biomarker in predicting long-term recovery of parathyroid function,”…
Severe COVID-19 could lead to the development of temporary hypoparathyroidism, a case report suggests. This report represents the third case of hypoparathyroidism secondary to severe COVID-19 infection noted in scientific literature. These rare instances, along with previous reports showing that the virus may worsen previously well-controlled hypoparathyroidism, highlight…
The dual transplant of a kidney and a parathyroid gland was safe and effective at restoring parathyroid hormone (PTH) levels and turning back kidney disease, in an 11-year-old girl with congenital hypoparathyroidism and renal failure, a case study reports. “Simultaneous living donation and transplantation of a kidney and a…
A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…
The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…
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