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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…

The European Commission (EC) has granted orphan drug designation to Ascendis Pharma’s experimental hormone replacement therapy TransCon PTH, currently in development for the treatment of hypoparathyroidism. Medicines…

A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…

Note: This story was updated on Nov. 12, 2020, to clarify key scientific concepts. A Phase 1 clinical trial of AZP-3601, an experimental therapy designed to induce a prolonged increase in blood calcium levels in people with hypoparathyroidism…

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…

People with thyroid cancer who develop hypoparathyroidism after surgery have reduced quality of life and worse symptoms than those without hypoparathyroidism, a study reports. The study, “Quality of life in patients with hypoparathyroidism after treatment for thyroid cancer” was published in The Journal of Clinical Endocrinology &…

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…

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…

Calcium deposits in the carotid arteries and brain are more common in people with permanent hypoparathyroidism who underwent thyroid removal surgery than in the general population, a study reports.  As such, calcifications should be monitored in this patient population, researchers said.

Having a greater than 65% drop in parathyroid hormone (PTH) levels on the first day after a total thyroid removal increases the likelihood of developing transient calcium deficiency, or hypocalcemia, according to a single-center study in China. People with hypocalcemia have lower than normal levels in their blood…