Webinar: Usher syndrome mini series – part one
This webinar covers key questions and topic areas including what Usher syndrome is, how to get a diagnosis, both clinical and genetic, and what will happen after this.
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Webinar: Usher syndrome mini series – part two
Professor Mariya Moosajee looks at the latest research, clinical trials and treatment prospects for people affected by Usher’s syndrome.
Sirius clinical trial – Exon 13 mutation of USH2A
Prof Mariya Moosajee at Moorfields Eye Hospital has asked us to share the message below about the clinical trial she is running. This trial is for a treatment that targets a particular section of the USH2A gene.
Aavantgarde Bio Ush1B study at Moorfields
Aavantgarde Bio Ush1B study at Moorfields
Look Forward – Summer 2024 – Issue 183
This edition includes exciting news about our Annual and Professionals’ Conferences. We hope as many of you as possible can join us in Manchester, or online, in September. You’ll also find updates about the latest research and a feature on one of our funded researchers, Dr Jörn Lakowski.
The Gene Team
Funding from Retina UK has allowed to team to explore the viability of gene therapy as a treatment for RP and related conditions, and build a pipeline of clinical trials.
Research update – ProQR
In February 2022, ProQR announced a disappointing outcome for their phase 2/3 trial of sepofarsen for Leber congenital amaurosis type 10 (LCA10).
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.
Sepul Bio begins clinical trial of ultevursen for USH2A-associated RP
Sepul Bio begins clinical trial of ultevursen for USH2A-associated RP