NICE approves first treatment on the NHS for an inherited retinal dystrophy
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 …
Continue reading "NICE approves first treatment on the NHS for an inherited retinal dystrophy"
ProQR Announces Clearance of IND to Start Clinical Trial
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 …
Continue reading “ProQR Announces Clearance of IND to Start Clinical Trial”
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
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. …
Continue reading “Southampton researchers embark on clinical trial for Stargardt disease”
Research in the news (April – June 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
‘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’ …
Continue reading "‘Encouraging’ early stage results from stem cell therapy trial"
Research in the news (February/March 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 …
Continue reading “Research in the news (February/March 2019)”
New developments in the search for treatments for Stargardt disease
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 …
Continue reading “New developments in the search for treatments for Stargardt disease”
Promising interim results for trial of potential new treatment for LCA10
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 …
Continue reading "Promising interim results for trial of potential new treatment for LCA10"
“First-in-class” treatment for vision loss in Usher syndrome takes a step towards clinical testing
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
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. …
Continue reading "First gene editing treatment moves towards clinical trials"
Gene therapy for RPE65 gene approved for use in EU
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 …
Continue reading “Gene therapy for RPE65 gene approved for use in EU”