Happy Rare Disease Week!
This week has been all about rare diseases. Today, we’re highlighting an article from a PW contributor with FAP, followed by a story describing some shortcomings of NICE. After that, we have updates on trials for patients with proteus syndrome and hypoparathyroidism.
Sit back and enjoy this week’s Editor’s Choice.
Laura shares a story about a condition that affects her family.
NICE is responsible for coming to the aid of rare patients in the UK, but not everyone is happy with how inaccessible medication can be.
A therapy should, ideally, have multiple studies that show its value. A new study reinforced the merit of a treatment for proteus syndrome.
Check out PaTH Forward, a global trial bringing home to the HP community!