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GENETIC DISEASES (*)'s subsections
Amyloidosis TransThyRetin Poly>*
AngioEdema *
Duchenne Muscular Dystrophy
Esophageal Atresia
Fabry Disease
FR. S. GD1>Treat.>EliGlustat Cap.
FR. S. LOPS>Treat.>AvalGlucosidase>
Gaucher Disease
IE. H. UCD>Treat.>GlycerolPhenyl>
JP. J. MPS II (Hunter Syndrome)>
JP. N. DMD>Treat.>Dystrophin pre->
MucoPolySaccharidosis VII
PhenylKetonUria *
Tyrosinemia Type 1 *
USA. A. Fragile X Syndrome DIAG
USA. A. PH Type 1>Treat.>Lumasiran>
USA. B. Achondroplasia>Treat.>CNP
USA. B. Gene Editing Technology

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