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Nearly 1 in 10 Americans is living with a rare disease. 95 percent of these diseases currently lack an FDA-approved treatment.
For many of these patients, access to treatments and breakthrough developments from clinical trials can mean the difference between life and death.Â
How has the pandemic impacted Americans living with rare diseases, particularly as clinical trials have been delayed? How are the scientific and medical communities prioritizing rare disease research amid competing health concerns? What is Congress doing to address the disruption of these scientific discoveries for rare disease patients?
On February 24, join The Hill to explore what’s at stake for the nearly 30 million Americans with rare diseases and the critical scientific research landscape they rely on.Â
At Harmony Biosciences, our goal is as much about delivering innovative therapies to help improve the lives of people with rare neurological diseases as it is about listening to them to discover what is often overlooked. By connecting empathy with science, we’re dedicated to addressing the primary and secondary disease symptoms that burden these communities. Learn more about our work with rare disease patients and organizations.