Phase 2 Trial of CMK389 Recruiting Adults With Pulmonary Sarcoidosis

A Phase 2 clinical trial is recruiting adults with chronic pulmonary sarcoidosis to investigate the safety and therapeutic potential of CMK389 in improving lung function. CMK389 is an investigational therapy, developed by Novartis, for the treatment of pulmonary sarcoidosis and atopic dermatitis. It was designed to inhibit interleukin 18,…

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

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.

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…