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HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

Symptoms of Uncontrolled Hypoparathyroidism Reduce Quality of Life for Patients, Caregivers, Survey Finds

Most people with uncontrolled, chronic hypoparathyroidism experience moderate-to-severe symptoms — even while on treatment — that lower their health-related quality of life (HRQoL), worsen their health status, and impact their ability to exercise or work, the results of a global survey show. The survey also found that greater symptom…

Phase 2 Study of Treatment for Hypoparathyroidism, TransCon PTH, Starts to Enroll Adults

A Phase 2 trial evaluating the efficacy and safety of TransCon PTH — designed to restore parathyroid hormone (PTH) levels within 24 hours — in people with hypoparathyroidism is now enrolling adults in North America and Europe. Called PaTH Forward, the study (NCT04009291) is planning to enroll about 40 patients with hypoparathyroidism who…