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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.

FDA Grants Fast Track Designation to PWS Potential Therapy LV-101

The U.S. Food and Drug Administration (FDA) has granted fast track designation to LV-101 (intranasal carbetocin), a treatment candidate for people with Prader-Willi syndrome (PWS). This designation is granted to promising therapies intended to treat serious diseases with unmet medical needs. It aims to quicken treatment development by…