Category: Research news

Study of substances in blood suggests choroideremia is not just an isolated eye disease

April 6, 2021

Choroideremia is a rare genetic condition resulting in progressive sight loss in men. Young boys suffer from difficulty with their night vision, followed by loss of their peripheral visual field …

Encouraging results from ProQR USH2A clinical trial

March 24, 2021

Biotechnology company ProQR has announced encouraging results from its phase 1/2 clinical trial of an innovative treatment for sight loss caused by faults in a specific section of the USH2A …

ProQR makes progress with potential treatment for LCA

January 18, 2021

Despite the challenges of the pandemic, researchers and pharmaceutical companies are still making progress towards delivering new treatments for inherited sight loss. Biotechnology company ProQR Therapeutics has announced that it …

Participants needed for Charles Bonnet syndrome (CBS) survey

June 8, 2020

Researchers at Moorfields Eye Hospital are conducting a short 5-15 minute survey about how the COVID-19 lockdown has affected people with Charles Bonnet syndrome (CBS). Responses to the survey will …

ProQR clinical trial for Leber’s Congenital Amourosis (LCA) 10

May 27, 2020

  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) …

Ask the expert

April 17, 2020

This service was offered for a limited time only and is no longer available. Would you like to speak with an expert who cares for lots of patients with inherited …

Positive early findings from ProQR trial of USH2A therapy

April 3, 2020

  Biotechnology company ProQR has announced encouraging results from its early analysis of the phase 1/2 trial of QR-421a, an innovative approach to treating sight loss caused by mutations in …

First clinical trial participant receives CRISPR-based therapy

March 4, 2020

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 …

First patient receives ground-breaking treatment

March 2, 2020

In January 23-year-old Jake Ternent became the first person with an inherited sight loss condition to be treated in the UK with Luxturna (voretigene neparvovec) for Leber congenital amaurosis (LCA). …

Gene therapy shows promise in tackling X-linked RP

February 28, 2020

Early results from clinical testing of a gene therapy to treat X-linked retinitis pigmentosa (XLRP) have shown partial reversal of sight loss in some patients. The treatment targets retinal degeneration …

Genetic testing: an essential topic for discussion with the IRD communtiy

February 13, 2020

As Luxturna reaches the clinic and other gene-specific therapies for inherited retinal disease get closer to the end of the development pipeline, it is becoming ever more important that affected …