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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.

Acthar Gel (repository corticotropin injection, or RCI) can improve sarcoidosis symptoms, a real-world study shows. Titled “Repository corticotropin injection in patients with advanced symptomatic sarcoidosis: retrospective analysis of medical records,” the study was published in the journal Therapeutic Advances in Respiratory Disease. Acthar Gel, marketed…

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…

The U.S. Food and Drug Administration (FDA) has granted the designation of breakthrough therapy to Ofev (nintedanib) for the treatment of patients with sarcoidosis and other types of progressive fibrosing interstitial lung diseases (ILDs). The breakthrough designation is given to medications that provide significant advantages over…