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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.

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

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…