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Displaying 551–560 of 898 results for “vision connection”
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Information for affected individuals and their families about the role of genetic counseling.
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First Patient Receives ProQR’s Emerging USH2A Therapy in Clinical Trial
Research NewsProQR, a developer of RNA therapies in the Netherlands, announced that the first clinical-trial participant has received its emerging treatment, which targets retinitis pigmentosa and Usher syndrome caused by mutations in exon 13 of the USH2A gene.
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The Foundation Fighting Blindness and Dr. H. James & Carole Free Collaborate to Combat AMD
Foundation NewsAge-related macular degeneration (AMD) is the leading cause of blindness for people over 50 years of age in developed countries.
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FDA Authorizes Clinical Trial for CRISPR/Cas9 Therapy for LCA 10
Research NewsEditas Medicine, a company developing gene-editing treatments, has received authorization from the US Food and Drug Administration to launch a clinical trial for its emerging CRISPR/Cas9 therapy for people with a mutation in the gene CEP290, which causes Leber congenital amaurosis 10 (LCA10). LCA causes severe vision loss or blindness at birth.
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ProQR Receives FDA Authorization to Launch Clinical Trial for USH2A Therapy
Research NewsProQR, a biotech in the Netherlands developing therapies for rare diseases, has received authorization from the US Food and Drug Administration to launch a Phase I/II clinical trial for QR-421a, its treatment targeting mutations in exon 13 of the USH2A gene.
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Acucela Enrolling Patients in Phase 3 Trial for Stargardt Disease Treatment
Research NewsThe Seattle biotech Acucela is now enrolling participants in its Phase 3 clinical trial for emixustat hydrochloride, an emerging oral drug for slowing vision loss in people with Stargardt disease, an inherited form of a macular degeneration.
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Beacon Stories
Kai Wang was 18 months old when he was diagnosed with the condition. His parents never imagined the extraordinary journey they would take with their son when they learned he had a condition that would render him blind.
