A Phase 2a clinical trial designed to test Galapagos‘ investigational oral therapy GLPG1690 in people with diffuse cutaneous scleroderma has completed enrolling participants, the company announced. GLPG1690 is a small molecule that targets and blocks autotaxin. This enzyme…
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.
Bob Saget will host the 42nd annual “Cool Comedy — Hot Cuisine” benefit of the Scleroderma Research Foundation (SRF) on Tuesday, Dec. 10. Saget, a well-known actor and a board member of SRF, will be joined by fellow comedians Michael Che, Jim Gaffigan and…
A single dose of an engineered antibody known as CD45-ADC can effectively eliminate disease-causing immune cells and ease the symptoms of scleroderma in mice, studies show. CD45-ADC is an example of Magenta Therapeutics‘ efforts to develop targeted antibody-drug conjugates (ADCs) to specifically remove disease-causing cells from the…
Health Canada has approved Ofev (nintedanib) as a treatment to slow the decline of pulmonary function in people with scleroderma-associated interstitial lung disease (SSc-ILD). The approval makes Ofev, by Boehringer Ingelheim, the first treatment for people with SSc-ILD available in Canada. It follows the therapy’s recent approval in the…
Cardiopulmonary exercise testing (CPET), along with an algorithm that considers both lung and heart functions, can help lower the unnecessary use of right heart catheterization to determine if a scleroderma patient has pulmonary arterial hypertension, a study reports. The study, “Cardiopulmonary exercise testing in a combined…
An advanced imaging technique, called ultra-high-frequency ultrasound (UHF-US), provides detailed information of all skin layers to better detect and characterize skin involvement in people with scleroderma, a pilot study reports. The ability of this technology to precisely measure damage in the dermis, the skin’s inner layer, may be…
People with diffuse cutaneous scleroderma (dcSSc) being treated with lenabasum continue to show improvement after more than two years of the therapy, an update from a Phase 2 extension study shows. The research, “…
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…
Black patients with scleroderma experience faster disease progression, higher prevalence of diffuse skin involvement, and higher mortality than Asian or white patients, a study reports. In addition, Asian patients were found to have earlier disease onset, faster disease course, and more lung involvement and pulmonary hypertension…
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