Prior to launching a clinical trial, the FDA must clear an Investigational New Drug (IND) application. This allows the drug to be distributed across state lines, which is important…
An experimental gene therapy program (called AXO-AAV-OPMD) being developed for the treatment of oculopharyngeal muscular dystrophy has been licensed with exclusive global rights to Axovant Sciences from Benitec Biopharma. There…
A new gene therapy treatment may be on the horizon for Oculopharyngeal Muscular Dystrophy patients. Find out more below, or keep up with the original story at musculardystrophynews.com. Oculopharyngeal Muscular…
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.
