Genetic testing: an essential topic for discussion with the IRD communtiy
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 …
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Latest update from ReNeuron stem cell trial
ReNeuron has announced an encouraging update on the progress of its Phase 1/2 trial of a stem cell-based therapy for retinitis pigmentosa, describing the most recent results as a “clear …
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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 …
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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 …
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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. …
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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’ …
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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 …
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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 …
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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 …
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“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 …