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Adeno-associated viral vector serotype 9 (AAV9) carrying both HEXA and HEXB

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Adeno-associated viral vector serotype 9 (AAV9) carrying both HEXA and HEXB is designated as an orphan drug by the US FDA on 7/17/2020, used in treatment of GM2 Gangliosidosis, manufactured / distributed by [[Taysha Gene Therapies


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