Prevail Therapeutics is the creator of PR001, which is a gene therapy used for the treatment of Gaucher disease. They expected to begin dosing in the first half of…
Oliver Sacks is beloved for his best-selling books that detail case studies of rare neurological disorders. Sacks created a niche when he combined his work as a neurologist with whimsical…
A team of cross-disciplinary researchers at Arizona State University (ASU) is working to develop a software which patients with neurological disorders may easily use to assess their disease progression. Through…
Copyscape score: 4% A common symptom of Rett Syndrome is apnea, which is a difficulty in breathing. This apnea has been a symptom that has had no treatment for a…
-150x150.jpg)
Researchers have learned that the majority of diseases have a genetic component. It is because of this fact that health professionals are stressing the importance of genetic testing and knowing…
Happy Thursday, Friends! It's getting cold outside! This week we're highlighting a new patient story from the Czech Republic on a younger patient diagnosed with a disease generally associated with…
According to a story from pharmanewsdaily.com, the gene therapy company Avrobio has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to AVR-RD-02, an…
According to a story from NBC News, Mitchell Herndon, a 19 year old from Missouri, recently passed away due to the progression of a rare genetic disease that is so…
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.
