Editor's Note: This article was originally published by Patient Worthy on 6/7/2017, and is being republished due to current NASH/MASH awareness efforts. If you would like to share your story…
I often find myself initially stuck on where to start when telling our story. I say “our” because that’s the truth of this rare disease journey: everyone who touches Natalie’s…
Go here to read Part 1, Part 2, and Part 3 of this series provided by TREND Community about Prader-Willi Syndrome. In January 2025, the TREND Community published another comprehensive report…
The treatment landscape for hemophilia B has taken a historic leap forward, as the first patient in the UK’s National Health Service (NHS) has received Hemgenix, a pioneering gene therapy…
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