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) …
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Ask the expert
Would you like to speak with an expert who cares for lots of patients with inherited sight loss conditions and has a detailed knowledge of the field? Consultant Ophthalmologist Dr …
Positive early findings from ProQR trial of USH2A therapy
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 a …
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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 …
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First patient receives ground-breaking treatment
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). …
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Gene therapy shows promise in tackling X-linked RP
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 …
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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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