After much hard work and dedication - the rare diseases community received amazing and encouraging news from NORD (National Organization for Rare Diseases)
https://rarediseases.org/senate-passes-landmark-21st-century-cures-act/
"We are so pleased to announce that today, the United States Senate overwhelmingly approved the 21st Century Cures Act (#CuresNow). Peter L. Saltonstall, President and CEO of NORD said, "After several years, we are excited to celebrate the Congressional enactment of (Cures). Once again we wish to convey our deep gratitude. This bill would have never passed without the hard work of patient advocates over the past several years." President Obama is expected to sign the bill into law later this week."
This is great news!! The new bill includes many provisions that will improve the discovery, development, and delivery of orphan therapies for rare disease patients.