GS-100 ⵉⴳⴰ ⵢⴰⵏ ⵓⵎⵙⵙⵓⴷⵓ ⵏ AAV9 ⵉⵜⵜⵓⵙⵎⵓⵏⵏ ⵉⵜⵜⵓⵙⵏⴼⵍⵏ ⴳ ⵓⵊⵉⵏ NGLY1 ⴳ ⵓⴼⴳⴰⵏ. ⵢⵓⵎⵥ ⵉⵙⵎ ⵏ ⵓⵙⴰⴼⴰⵔ ⵏ ⵡⴰⵔⵔⴰⵡ ⵙⴳ ⵜⵎⵀⵍⴰ ⵏ ⵡⵓⵜⵛⵉ ⴷ ⵓⵙⴰⴼⴰⵔ ⵏ ⵎⵉⵔⵉⴽⴰⵏ ⴷ ⵜⵙⵏⵓⵔⴰⵢⵜ ⵏ ⵉⵙⴰⴼⴰⵔⵏ ⵏ ⵓⵕⵓⴱⴱⴰ (EMA). ⵢⵓⵎⵥ ⵓⵙⵉⵊⵊⵉ ⴰⵡⴷ ⴰⵙⵎ ⵏ ⵡⴰⵟⵟⴰⵏ ⵏ ⵉⵎⵥⵢⴰⵏ ⵉⴷⵔⵓⵙⵏ ⵏ FDA ⴳ 2021 ⴷ ⵓⵙⵎ ⵏ ⵓⵙⵎⵓⵜⵜⴳ ⴰⵎⵣⵣⵓ ⴳ ⵓⵙⴳⴳⵡⴰⵙ ⵉⵣⵔⵉⵏ.
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