News

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…

Aeterna Zentaris, in collaboration with the University of Sheffield, in the U.K., selected AEZS-150 as its lead candidate for primary hypoparathyroidism and will begin formal preclinical studies to assess its therapeutic potential. Data from this preclinical program may support the future launch of the first clinical trial to…

People with chronic hypoparathyroidism have a significantly higher risk of kidney stones compared with patients without chronic disease, a large study finds. These patients also have a much greater risk of experiencing calcium deposits or calcification — called nephrocalcinosis…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

Levels of parathyroid hormone (PTH) accurately predicted hypoparathyroidism-related calcium deficiency following surgery to remove the thyroid gland, a study reported.  A PTH cut-off value was established to identify those at risk, who were started on calcium and vitamin D supplements. Such prompt treatment eased symptoms…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

People who develop chronic hypoparathyroidism following surgery to remove the thyroid gland have significantly poorer quality of life than those whose surgery does not permanently damage the neighboring parathyroid glands, a Danish study shows. Notably, hypoparathyroidism’s negative affect on life was independent of the drop in thyroid-related hormones (hypothyroidism) due…

An interruption in the supply of Natpara 100 micrograms (mcg) could begin as early as this week and last for about one year, Takeda Pharmaceuticals announced in an open letter to the Hypoparathyroidism Association and to the broader hypoparathyroidism patient and healthcare community. “As part of…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

A single dose of AZP-3601 — Amolyt Pharma’s experimental therapy for hypoparathyroidism — resulted in a significant, sustained increase in blood calcium levels for at least 24 hours in healthy volunteers, according to interim data from a Phase 1 trial. These early findings support the therapeutic potential of…