Alois Alzheimer, a clinical psychiatrist and expert in the study of the nervous system, reported a disease process of the cerebral cortex. The year was 1906. We are approaching July…
BioMan Pharmaceuticals has just announced that vosoritide has just received a positive opinion from the Committee for Medicinal Products in Europe, which means marketing authorization is recommended. Further, it has just…
The FDA grants designations, such as the Rare Pediatric Disease and Orphan Drug designations, to aid in the drug development process. Recently, LEXEO Therapeutics has received these two designations for…
The very first corneal allogenic intrastromal ring segment surgery (CAIRS) for keratoconus has just been completed in Australia. It was completed at Queensland Eye Institute by David Gunn. This procedural…
According to Muscular Dystrophy News, new research suggests that the cholesterol metabolic pathway could offer a potential therapeutic target for patients with Duchenne muscular dystrophy (DMD). Following microRNA analysis, researchers…
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…
When I think of anxiety, I think of the movie, Gremlins (1984). For those of you who have seen this movie, you know that feeding a gremlin after midnight turns…
Patient Worthy was a media partner at this year's NORD Living Rare, Living Stronger Patient and Family Forum. This is an annual event hosted by the National Organization for Rare…
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