A groundbreaking study has demonstrated that gene therapy can successfully restore hearing in patients with hereditary deafness, offering new hope for individuals affected by genetic forms of hearing loss. The…
As reported by HD Buzz, a significant step forward for Huntington’s disease (HD) research, Roche has announced that the first patient has been dosed in its new gene therapy clinical…
Researchers have discovered a potential “window” whereby babies born with inherited diseases may be treated with gene therapy sent directly into their circulatory systems. The window consists of circulating stem…
An article titled “Accelerated Approval as the New Norm in Gene Therapy for Rare Diseases” from DIA Global Forum digital magazine’s May 2025 issue explores the evolving landscape of regulatory…
Have you ever wondered how a rare disease can take control of a person’s life? The deteriorating health, being unable to receive an accurate diagnosis, and more importantly finding an…
© Copyright 2024 Patient Worthy
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.
