Posted by Michael Wonder on 20 Jul 2020
Rare disease drove two women on a mission to change FDA drug review process
19 July 2020 - Melissa Goetz and Lindsey Sutton are campaigning to improve the US drug review process for rare diseases.
When Californian Lindsey Sutton was an infant, her blood was the colour of cherry Pepto Bismol. Doctors quickly discovered it was highly saturated with fat.
At 5 weeks old, she was diagnosed with a rare genetic disease called familial chylomicronaemia syndrome, or FCS -- a disorder that would lead to lifelong bouts of severe abdominal pain, liver problems and pancreas inflammation.
Years later in New York, Melissa Goetz found out her weeks-old daughter also had the disease, which causes a buildup of fats in the blood and can cause potentially fatal problems with the pancreas. Goetz's baby would soon be hospitalised with pancreatitis, a liver infection and a kidney infection.
