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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…

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…