Diagnosis and beyond
Making yourself aware of the what you are entitled to, and understanding the diagnosis pathway for inherited sight loss, will put you in a stronger position to seek better support.
Search
Search results
Frequently asked questions
The following questions are often asked by people contacting Retina UK.
ProQR makes progress with potential treatment for LCA
Despite the challenges of the pandemic, researchers and pharmaceutical companies are still making progress towards delivering new treatments for inherited sight loss.
ProQR clinical trial for Leber’s Congenital Amourosis (LCA) 10
Biopharmaceutical company ProQR is conducting a Phase 2/3 clinical trial (Illuminate) to measure the safety and effectiveness of RNA therapy, Sepofarsen which aims to treat Leber's Congenital Amourosis (LCA) 10.
First clinical trial participant receives CRISPR-based therapy
An American living with Leber congenital amaurosis 10 (LCA10) has become the first clinical trial participant in the world to receive a CRISPR gene editing treatment in vivo (inside the body).