In the European Union (EU), Orphan designation, or Orphan Medicinal Product designation, is granted to drugs or biologics intending to treat, diagnose, or prevent a rare, chronically debilitating, or…
In March 2022, T. Kavinraj began experiencing a number of worrying health symptoms. He had a splitting headache and could not stop vomiting. In fact, his condition was so bad…
In the United States, the Food and Drug Administration (FDA) grants Orphan Drug designation to drugs or biologics which intend to treat, diagnose, or prevent rare conditions. “Rare” conditions are…
Ten years ago, a biochemist was seeking investors for her new company that was developing mitochondrial-based medicines. The Israeli-born embryologist observed how the blending of mitochondria from one egg…
This year, the 13th annual Moebius Syndrome Awareness Day (MSAD) will take place on Tuesday, January 24. Moebius Syndrome Awareness Day, as the name suggests, is an event to celebrate…
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.
