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Five-year-old Olivia Riley of Arizona suffers from an extremely rare and fatal genetic brain disorder called metachromatic leukodystrophy (MLD). She..

FDA red tape kept effective treatment an ocean away. While reading Judy Stecker’s “The FDA Could Help Save My Son From..

Right to Try has been a literal lifesaver for many Americans with deadly diseases such as cancer and amyotrophic lateral sclerosis (ALS,..

Last week, President Joe Biden commemorated the 60th anniversary of former President John F. Kennedy’s historic moonshot speech in which he..

This is the third piece in a four-part series about Right to Try at the federal and state levels and..

You wouldn’t know it from examining today’s drug approval system, but there was once a time when federal regulations empowered..

It’s a revolutionary reform born out of an appalling reality: Dying Americans are routinely blocked from potentially lifesaving treatments unless..

  The numbers are staggering. Approximately 7,000 rare diseases affect between 25 and 30 million, according to the National Institutes..

There’s a new treatment for a rare disease. It’s custom-made for you, based on your own genetic profile. It offers..

In March of 2020, my family got the news that would forever change all of our lives: My daughter Olivia,..

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