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Leber optic atrophy
- ... treat a small number of diseases, including an eye disorder called Leber congenital amaurosis and a muscle disorder called spinal muscular atrophy . Many more gene therapies are undergoing research to ...
- ... are available for conditions that include a rare eye disorder called Leber congenital amaurosis , a form of skin cancer known as melanoma , and a genetic muscle condition called spinal muscular atrophy . Other genetic therapies have been approved for blood ...
