According to a story from keloland.com, Westin Cuka, age six, was first diagnosed with neurofibromatosis, a rare disorder, three years ago. Since then, the family has traveled on multiple occasions…
Biotechnology company Talaris Therapeutics, Inc. submitted an Investigational New Drug (IND) application to the FDA for their novel cell therapy treatment FCR001. As per a press release, the FDA…
Rare pediatric disease designation has just been granted to Rhythm Pharmaceuticals for their investigative therapy called setmelanotide. This drug is being investigated as a therapeutic option for two conditions- leptin…
In a July 9 press release, the Familial Chylomicronemia Syndrome (FCS) Foundation announced its support for a bill introduced by Congressman Paul Tonko (D-NY) and David B. McKinley (R-WV).…
Within the medical realm, clinical trials play a crucial role in understanding disease progression, medication safety and efficacy, and patient outcomes. Recently, PTC Therapeutics began a Phase 1 clinical…
According to Parkinson's News Today, the Parkinson's Foundation has released data from the PD GENEration study that shows that genetic testing is feasible for the larger Parkinson's community. As around…
The FDA has recently approved pembrolizumab, which is a treatment for colorectal cancer, specifically MSI-H and dMMR colorectal cancer. This approval comes after the release of positive results of the…
Each year, June 13th is celebrated as Gastrointestinal Stromal Tumors (GIST) Awareness Day. The ultimate goal of awareness days for rare diseases is to help inform both the general public…
As COVID-19 grips the world, scientists are chasing after it to find the full spectrum of effects as it interacts with human biochemistry. While known for its characteristic cough and…
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.
