Author Archives: Marisa Wexler MS

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…

Ascendis Pharma‘s TransCon PTH can safely treat hypoparathyroidism, reducing the need for other therapies and improving patients’ quality of life, an extension period of the PaTH Forward Phase 2 trial shows. “Today we announced preliminary clinical results which demonstrate that TransCon PTH can potentially…

TransCon PTH, an investigational therapy being developed by Ascendis Pharma, safely normalized calcium and phosphate levels, and reduced the need for other treatments in adults with hypoparathyroidism, data from the PaTH Forward Phase 2 trial demonstrate. The data were presented by Ascendis…

A novel approach for the production of parathyroid-like cells from stem cells — cells from which all others with specialized functions are generated — could help find new treatments for hypoparathyroidism, a study suggests. The study, “Differentiation of parathyroid hormone expressing cells from human pluripotent…