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Leber Congenital Amaurosis (LCA)
LCA is a group of inherited retinal diseases causing blindness or severe vision loss in early childhood.
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Choroideremia is an inherited retinal disease that causes progressive vision loss due to degeneration of the choroid, the retinal pigment epithelium or RPE, and the photoreceptors.
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Often diagnosed in childhood or adolescence, BBS is an inherited disease causing progressive loss of night and peripheral vision from retinitis pigmentosa. BBS can also cause a number of other symptoms and problems including: obesity, extra fingers and toes, kidney disease, and developmental disabilities. Symptoms vary from person to person.
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Best disease is an inherited retinal disease, which causes macular degeneration and loss of central vision, visual acuity, and color perception.
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Achromatopsia is an inherited retinal disease causing loss of visual acuity as well as central and color vision and vision in lighted conditions.
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Jan 23, 2018
Ophthotech Launching Human Study of Emerging Therapy for Stargardt Disease
Ophthotech, a biopharmaceutical company developing therapies for eye diseases, has enrolled the first patient in its Phase 2b clinical trial of Zimura® for people with Stargardt disease caused by mutations in the gene ABCA4.
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Jan 18, 2018
The Assistance Fund Opens New Program for Inherited Retinal Diseases
The Assistance Fund, an independent charitable patient assistance foundation that helps patients and families facing high medical out-of-pocket costs, today launched a new program that offers financial support for individuals with inherited retinal diseases.
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Jan 17, 2018
Clinical Trial to Launch for System Combining Optogenetics and Eyewear
The French biotech GenSight Biologics has received regulatory authorization in the UK to launch the PIONEER Phase 1 \ 2 clinical trial for its GS030 system — a light-sensing gene therapy (optogenetics) coupled with eyewear, which enhances visual stimulation.
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Often diagnosed in childhood or adolescence, retinitis pigmentosa (RP) is an inherited retinal disease causing progressive loss of night and peripheral vision. The condition often leads to legal and sometimes complete blindness.
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Jan 9, 2018
A Retinal Research Nonprofit Paves the Way for Commercializing Gene Therapies
AN EMERGING, vision-restoring gene therapy for a devastating retinal disease is poised for Food and Drug Administration (FDA) approval. If it gets the regulatory nod, it will be the first gene therapy to receive FDA approval for the eye or an inherited condition.