The U.S. Food and Drug Administration has granted fast track designation to encaleret, an investigational therapy for autosomal dominant hypocalcemia type 1, called ADH1, a rare genetic form of hypoparathyroidism. Encaleret is designed to help normalize the calcium levels of ADH1 patients by blocking the calcium-sensing receptor…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…
From buying virtual lemonade drinks to participating in a town hall meeting, the hypoparathyroidism community is poised to mark World Hypopara Awareness Day, observed each June 1. Patients, caregivers, and advocates worldwide are invited to participate in the event aimed at heightening awareness among the general public, as well…
Calcium deposits — known as calcifications — in the kidneys and higher-than-normal levels of calcium in urine are common in people with chronic hypoparathyroidism, according to a small Danish study. Notably, the presence of these calcifications was neither significantly associated with the levels of calcium in the urine or…
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…
Get regular updates to your inbox.
