According to a story on pharmaphorum.com, the UK's National Institute for Health and Care Excellence (NICE) has given a positive opinion for the drug vutrisiran (marketed as Amvuttra) as a…
Amber Freed has long been a tireless advocate for the rare disease community. After two years of IVF, she became pregnant with her two children: Maxwell and Riley. About four…
Cyndi Strzynski was always a bubbly, active little girl. Even though she has been facing an immense challenge, she still tries to keep a smile on her face at all…
In 2020, Lily Tyson, aged 12 at the time, was diagnosed with a rare chromosomal disorder called Turner syndrome. More recently, Lily began experiencing various eye issues: redness, pain, blurred…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
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