Marisa Wexler, MS, senior science writer —

MBX 2109, an experimental hormone replacement therapy that MBX Biosciences is developing for hypoparathyroidism, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The FDA awards this designation to therapies intended to treat rare diseases, defined as those that affect less than 200,000 people…

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…

A new study out of the Netherlands has found a high incidence — 15% — of long-term hypoparathyroidism in patients after surgery on the thyroid gland. “These data provide a better understanding of the magnitude of this postoperative complication and enable more realistic information for patients who are undergoing…

AZP-3601, an investigational treatment for chronic hypoparathyroidism, was able to safely increase blood calcium levels in healthy volunteers in a Phase 1 clinical trial, top-line data show. “We are pleased with these clinical trial results for AZP-3601 … as they support a potential therapeutic profile that we believe can address…

Typically, it takes nearly three months before people newly diagnosed with chronic, post-surgical hypoparathyroidism arrive at a stable dose of calcium and vitamin D supplements, a study reported, rather than several weeks. But many patients in this study also experienced abnormally high or low calcium levels even after a stable dose…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Ascendis Pharma is requesting regulatory permission to open a Phase 3 clinical trial in Japan, part of its program to assess TransCon PTH as a hormone replacement therapy for adults with hypoparathyroidism at sites worldwide. Ascendis filed a clinical trial notification (CTN) for what it will call the PaTHway Japan…

People with hypoparathyroidism in Argentina, particularly those with kidney problems and high levels of phosphate in the blood, frequently experience inadequate disease control, a study found. The study is the first to analyze hypoparathyroidism in Latin America, researchers said. Titled “Hipopara-Red, Real Life Experience in 322…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…