Together we can bring hope for sight & sound

Our board member Deirdre, and Prof Keegan, recently appeared on national TV on Ireland:AM, to raise awareness of Usher syndrome and Usher Syndrome Ireland. You can watch back by searching episode number:128 on Virgin Media Player.

 Bringing hope to the Usher syndrome community, ProQR has recently begun dosing its first patients in its Phase 2 /3 Sirius trial for people with Usher syndrome due to mutations in exon 13 of the USH2A gene. This trial is testing whether its RNA therapy, ultevursen, is effective at halting vision loss. and whether it is safe and well-tolerated. More information can be found here. 

  Nacuity has begun its Phase 1/2 clinical trial of Nacuity’s proprietary NPI-001 tablets, the SLO-RP Study, and are now enrolling patients with RP associated with Usher syndrome. This therapy focuses on the treatment of ocular oxidative stress in the retina which they believe may potentially slow down the degeneration of the photoreceptors cells as experienced by patients with RP associated with Usher syndrome. Nacuity expects to report interim safety results from this study during the third quarter of 2022 and first efficacy results second quarter 2023. More information can be found here.