Written by Lily Sander By the time I was age four, I started experiencing unexplained symptoms such as severely turned in feet, which caused extreme pain and mobility issues. Terrified,…
On May 18, 2023, Market Watch reported that rucosopasem manganese received Orphan Drug designation. Developed by clinical stage biopharmaceutical company Galera Therapeutics (“Galera”), rucosopasem manganese is largely being developed…
Within the Phase 3 APHENITY study, researchers worked to determine the efficacy of sepiapterin in both adults and children with phenylketonuria (PKU). Formerly known as PTC923, sepiapterin was developed…
In 2014, the National Heart, Lung, and Blood Institute released updated clinical guidelines in reference to the care of children with sickle cell anemia. The updated guidelines recommended the use…
The damage caused by cocaine can sometimes be misdiagnosed as a rare disease: granulomatosis with polyangiitis. Cocaine use has been increasing in America and the UK, resulting in serious…
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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