Category: Research news

NICE approves first treatment on the NHS for an inherited retinal dystrophy

September 4, 2019

Today the National Institute for Health and Care Excellence (NICE) has recommended  Luxturna (voretigene neparvovec) for use in the NHS in England, making it the first available treatment for an …

ProQR Announces Clearance of IND to Start Clinical Trial

August 14, 2019

ProQR Therapeutics announced on Monday 12 August that the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for QR-1123, which is the first investigational medicine to …

Awaiting funding decision for Luxturna

In the last issue of Look Forward, we reported that the gene therapy Luxturna had won European regulatory approval for the treatment of a particular type of inherited sight loss. …

Southampton researchers embark on clinical trial for Stargardt disease

July 3, 2019

Researchers in Southampton are recruiting people living with Stargardt disease for the clinical trial of a new drug called Remofuscin that could limit the sight loss associated with this condition. …

Research in the news (April – June 2019)

July 1, 2019

There have been some exciting research developments from around the world in the last few weeks. Here are two stories that we have already shared via Facebook and Twitter. Calming …

‘Encouraging’ early stage results from stem cell therapy trial

April 15, 2019

‘Encouraging’ early stage results have been reported from an ongoing clinical trial for a stem cell therapy for retinitis pigmentosa. Retina UK has welcomed the news, while stressing ‘cautious optimism’ …

Research in the news (February/March 2019)

March 29, 2019

There have been some exciting research developments from around the world in the last few weeks. Here are some stories from our colleagues at other organisations, which we have already …

New developments in the search for treatments for Stargardt disease

January 31, 2019

Researchers funded by Retina UK have contributed to the early development of potential new treatments for Stargardt disease, with their findings recently published in the journal Genetics in Medicine. Stargardt …

Promising interim results for trial of potential new treatment for LCA10

December 18, 2018

Highly promising interim results from an early phase clinical trial of a potential new treatment for Leber’s congenital amaurosis 10 (LCA10), caused by mutations in the CEP290 gene, have been …

“First-in-class” treatment for vision loss in Usher syndrome takes a step towards clinical testing

December 6, 2018

A potentially transformative new treatment for vision loss in Usher syndrome type 2 has been given the go-ahead for initial clinical testing in a small number of adults living with …

First gene editing treatment moves towards clinical trials

December 4, 2018

The US Food & Drug Administration (FDA) has given the go-ahead for clinical testing of what could be the first gene editing treatment to be used in inherited eye disease. …

Gene therapy for RPE65 gene approved for use in EU

November 23, 2018

The gene therapy Luxturna has won European regulatory approval for the treatment of inherited retinal dystrophy caused by mutations in the RPE65 gene. This milestone decision was made by the …