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Behold the possibilities of emerging therapies

Behold the possibilities of emerging therapies

Today, numerous clinical trials are either completed or underway for the treatment of different types of inherited retinal diseases, such as retinitis pigmentosa, X-linked retinitis pigmentosa (XLRP), Leber congenital amaurosis (LCA), achromatopsia (ACHM), Usher syndrome and Stargardt disease. These trials explore the potential of gene therapy and several other technologies.

After receiving your results, your genetic counsellor and eye specialist may recommend extra steps to find out if you are eligible for a clinical trial or approved therapy.

Learn more about ongoing trials here.

Science is advancing the future of inherited retinal disease treatment

Many gene therapy innovations are now being studied. Each therapy (below) is designed to affect genes associated with inherited retinal disease in a specific way.

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Gene augmentation therapy

works by replacing mutated genes with new normal copies of that gene

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Gene editing

corrects the gene variant directly, instead of delivering a new gene

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RNA (ribonucleic acid) editing

fixes the instructions required to create proteins

See what’s possible when you test or retest.

Ask your eye specialist about genetic testing today.

More answers lie in our genes

While you can’t change what you inherit, you may find answers that can help put you in control of your future.

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5 simple steps to test

Know what to expect before, during, and after your genetic test or retest.

References

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Cancer.Net. Amyloidosis: Symptoms and Signs. Accessed May 26, 2022.
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Mayo Clinic. Amyloidosis overview: symptoms and causes. Accessed May 26, 2022.
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National Organization for Rare Disorders. Amyloidosis. Accessed May 26, 2022.
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D'Errico S, Mazzanti A, Baldari B, Maiese A, Frati P, Fineschi V. Sudden death in lambda light chain AL cardiac amyloidosis: a review of literature and update for clinicians and pathologists. Int J Clin Exp Pathol. 2020 Jul 1;13(7):1474-1482. PMID: 32782665; PMCID: PMC7414507.
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Mayo Clinic. Amyloidosis diagnosis and treatment. Accessed August 20, 2020.
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Lousada I, Comenzo RL, Landau H, et al. Light chain amyloidosis: patient experience survey from the Amyloidosis Research Consortium. Advances in Therapy. 2015;32(10):920-928.
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Merlini G, Dispenzieri A, Sanchorawala V, et al. Systematic immunglobulin light chain amyloidosis. Nature Review Disease Primers. 2018;4(1):38.
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