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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Myeloma UK AL amyloidosis Essential Guide. Accessed May 30, 2022.
Merlini G, Palladini G. Light chain amyloidosis: the heart of the problem. Haematologica. 2013;98(10):1492.
NHS. Amyloidosis. Accessed May 26, 2022.
McCausland KL, White MK, Guthrie SD, et al. Light chain (AL) amyloidosis: the journey to diagnosis. The Patient-Patient-Centered Outcomes Research. 2018;11(2):207-16.
Stanford Health Care. AL (Primary) Amyloidosis. Accessed May 26, 2022.
John Hopkins Medicine. What is amyloidosis?. Accessed May 26, 2022.
Amyloidosis Foundation. AA Amyloidosis. Accessed May 26, 2022.
Amyloidosis Foundation. Hereditary Amyloidosis. Accessed May 26, 2022.
Amyloidosis Foundation. Wild-type Amyloidosis. Accessed May 26, 2022.
Mayo Clinic. Amyloidosis. Accessed May 26, 2022.
Myeloma UK. AL amyloidosis. Accessed May 26, 2022.
Merlini G, Dispenzieri A, Sanchorawala V, et al. Systematic immunglobulin light chain amyloidosis. Nature Review Disease Primers. 2018;4(1):38.
Bahlis NJ, Lazarus HM. Multiple myeloma-associated AL amyloidosis. Bone Marrow Transplantation. 2006;38;7-15.