The Phase 3 PLEO-CMT clinical trial evaluated the safety, efficacy, and tolerability of PXT3003 for patients with Charcot-Marie-Tooth disease type 1A (CMT1A). Altogether, 323 people enrolled. During the trial, researchers…
Ryan Smith has always been tenacious in pursuing his goals. One of his most important goals? Supporting his family and his community. As a lieutenant with the Stillwater Fire Department,…
There are a number of different mosquito-borne illnesses. But sometimes it can be difficult to make an accurate diagnosis when these illnesses – and many others – share similar symptoms…
The 1st International Workshop on Myelodysplastic Syndromes was held this year in Miami, FL from June 24-26. This in-person event was a convergence of researchers and clinicians to discuss advances…
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…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
