When Brett Devloo was fifteen years old, he lost his sight. It happened, he feels, suddenly; one minute, Brett was reading and writing notes in his history class and…
For five years, 20-year-old Christopher Backlund lived with his grandfather, with whom he had a strong and meaningful bond. Unfortunately, Christopher's grandfather, Mitchell, passed away in Februay 2021. As Christopher…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. These are defined as conditions affecting under 200,000 Americans.…
Recently, research and development (R&D) company Neurophth Therapeutics announced that the FDA granted Orphan Drug designation to its therapy NR082. Also known as rAAV2-ND4 or NFS-01 project, NR082 is…
After three years, patients with Leber hereditary optic neuropathy (LHON) who had been treated with LUMEVOQ experienced sustained safety and efficacy. According to a press release from GenSight Biologics,…
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.
