Belite Bio – Phase 2/3 Trial for Adolescents with Stargardt disease
The latest research news piece from Retina UK.
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Volunteer Impact Report
Our volunteers are highly valued members of the Retina UK team and their roles are essential to help support and enable people affected by inherited sight loss to live fulfilling lives.
AAVantgarde Completes Enrolment in Phase 1/2 Clinical Trial for Usher Syndrome Type B
The latest research news piece from Retina UK.
Ocugen announce Phase 3 enrolment completion for OCU400, the largest gene agnostic therapy trial for RP
Biotechnology company Ocugen has announced the enrolment completion of their phase 3 clinical trial for OCU400.
x-linked inheritance
X-linked inheritance means that the faulty gene is located on the X chromosome, the larger of the two sex chromosomes.
The molecular map of congenital stationary night blindness – the science of seeing in the dark
A recently published study has provided the first molecular map of the mutation-specific changes that occur in the retina which lead to Congenital Stationary Night Blindness.
Gene Therapy improves functional measures in XLRP
Beacon Therapeutics announced positive results from their phase 2 gene therapy trial for X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.
Significantly worse vision found in female carriers of X-linked RP and Choroideremia
It was previously believed that female carriers of X-linked inherited retinal diseases (IRDs) like X-linked retinitis pigmentosa (RP and Choroideremia) remained unaffected by sight loss.
SB-007 update: dose-expansion portion of SpliceBio’s phase 1/2 trial underway with first participant dosed at Oxford
SpliceBio has begun the dose-expansion portion of their phase 1/2 clinical trial of SB-007, a dual adeno-associated viral vector gene therapy for Stargardt disease.
Non-coding, but not non-important: the hidden genes behind retinitis pigmentosa
A new study by researchers from Radboud University Medical Centre and the University of Basel has uncovered new genetic causes of Retinitis Pigmentosa (RP) that were once overlooked.