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Congenital disorder
- ... disease Beta thalassemia Bloom syndrome Canavan disease PMM2 -congenital disorder of glycosylation , also called PMM2 -CDG Cystic fibrosis ... bifunctional protein deficiency Dihydrolipoamide dehydrogenase deficiency Familial ... storage disease type I Hereditary fructose intolerance ...
- ... of diseases, including an eye disorder called Leber congenital amaurosis and a muscle disorder called spinal muscular atrophy . Many more gene therapies ...
- ... available for conditions that include a rare eye disorder called Leber congenital amaurosis , a form of skin cancer known as ...
- ... of gene therapies, including therapies to treat inherited disorders like spinal muscular atrophy and Leber congenital amaurosis . Learn how to cite this page