Encouraging results from ProQR USH2A clinical trial
Biotechnology company ProQR has announced encouraging results from its phase 1/2 clinical trial of an innovative treatment for sight loss caused by faults in a specific section of the USH2A gene.
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Biotechnology company ProQR has announced encouraging results from its phase 1/2 clinical trial of an innovative treatment for sight loss caused by faults in a specific section of the USH2A gene.
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X-linked inheritance means that the faulty gene is located on the X chromosome, the larger of the two sex chromosomes.
Biotechnology company ProQR has announced that its clinical trials of sepofarsen for Leber congenital amaurosis type 10 and ultevursen for USH2A-mediated retinitis pigmentosa will be wound down with immediate effect.
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.
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 families can access a genetic diagnosis, potentially opening up choices around treatment and clinical trial participation.