C. difficile is a bacterial infection that impacts the gastrointestinal system. It can lead to weight loss, diarrhea, dehydration, and even the loss of blood platelets. Megan Newman has had…
Lyme disease is a vector-borne disease spread through ticks. It is most common in the Northeast, North-Central, and mid-Atlantic portions of the country, but is notably absent in the Southeast.…
Growing up, I was always told that oral health related directly to regular health. But now, a study from researchers at the NYU College of Dentistry and Weill Cornell Medicine…
Sawyer Burch is a four-year-old boy from the Nashville, Tennessee area who was born with trifunctional protein deficiency, a rare disorder that takes the body's ability to utilize certain types…
Passage Bio has recently announced that Orphan Drug Designation has been granted by the European Commission for PBKR03, a gene therapy for Krabbe Disease. This condition has yet to have…
In mid-April, Kantar Health and the International Gaucher Alliance (IGA) shared the launch of a rare disease registry developed by the partnership. The registry, known as GARDIAN, will explore Gaucher…
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…
Earlier this year, WP1066, a p-STAT3 inhibitor, received three separate Rare Pediatric Disease designations for the treatment of medulloblastoma, atypical teratoid rhabdoid tumor, and diffuse intrinsic pontine glioma. Now,…
Clinical trials can be beneficial in many ways. For one, these trials show researchers whether a treatment’s impact is durable and sustained. Determining this often requires some follow-up period. According…
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