In the past, there have not been any available treatments to tackle cholestatic pruritus, or a severe and unrelenting itch, for patients with liver diseases such as Alagille syndrome. However,…
NOTA, The Network of Tyrosinemia Advocates, held their 4th Annual NOTA Experience, a family conference in September, despite the extraordinary constraints of time, a pandemic, transportation challenges, and the special…
Clinical trials are important to understanding both more about certain conditions and more about the safety, efficacy, and tolerability of drugs designed to treat these conditions. Currently, biopharmaceutical company AMO…
It's a beautiful thing when a community pulls together to support one of its members. This is exactly what the Madeira community is doing for 16-year-old Diego Ramirez. He recently…
Lytle, Texas is a small town located outside of San Antonio. Recently, the entire community came together to support a young boy living with autosomal recessive polycystic kidney disease (ARPKD).…
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…
The FDA created its Orphan Drug Act to incentivize drug developers to develop solutions for patients with rare or life-threatening conditions. Now, Orphan Drug designation is granted to drugs or…
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