Gene therapy treatment Luxturna accepted for use in Scotland
Retina UK is delighted that the Scottish Medicines Consortium (SMC) has accepted the gene therapy Luxturna (voretigene neparvovec) for ongoing use by the NHS in Scotland.
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Why genes matter
Genes exist inside all the cells that make up your body. They contain genetic code (DNA) and provide instructions for constructing proteins, which are the building blocks of our bodies and perform a huge variety of roles.
Inheritance patterns
Your doctor or genetic counsellor will spend some time asking about other people in your family to try to work out the way in which your particular faulty gene has been passed down through the generations – this is referred to as the inheritance pattern.
Look Forward – Winter 2022 – Issue 178
The cover of this edition celebrates the success of our amazing London Marathon team. Together they have raised more than £43,000.
Meira GTx announces positive data from phase clinical trial
Biotechnology company MeiraGTx has announced encouraging news from its phase 1/2 clinical trial of botaretigene sparoparvovec (previously known as AAV-RPGR), a gene therapy aimed at X-linked retinitis pigmentosa caused by faults in the RPGR gene.
Frequently asked questions
The following questions are often asked by people contacting Retina UK.
Exciting developments for new treatment research
In recent years there has, understandably, been a lot of excitement around cutting-edge therapies that target the specific genetic faults underlying inherited sight loss.
Research brings hope for the future
“All the promising research makes you feel like there is light at the end of that very dark tunnel after all.”
Fundraising FAQs
Fancy fundraising for Retina UK? Here’s a few pointers to help get you started.
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 particular section of the USH2A gene.