Family of Twins with Rare and Fatal Cholesterol Disease To Seek FDA Approval To Deliver Cyclodextrin Into the Brain

The tagline of the new movie, Extraordinary Measures, about John Crowley’s fight to save his children from the rare and fatal Pompe disease is….Don’t Hope For a Miracle – Make One! We are now working on making our own miracle  – a new compassionate use investigational new drug (IND) protocol and application that we plan […]

Extraordinary Measures Movie Is A National Healthcare Wake Up Call

Extraordinary Measures, a new movie centered on the efforts of John and Aileen Crowley to find a researcher who might help find a cure for their two children’s rare genetic disease (Pompe Disease), will hit theaters on January 22. I can’t wait to see this movie!  The trailer sounds eerily familiar as we are working around […]