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  "authorName": "Taysha Gene Therapies",
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  "notes": "Includes a molecular diagram of glycogen branching and a therapeutic component breakdown (AAV9 + hGBE1 + Intrathecal delivery).",
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      "text": "APBD is caused by reduced glycogen branching enzyme (GBE1) activity\nGBE1 mutations lead to uncontrolled glycogen elongation and the formation of polyglucosan bodies\nSymptoms include sensory loss in the legs, progressive muscle weakness, gait disturbances, urinary difficulties, and mild cognitive impairment\nThe estimated prevalence of APBD is 1,000 patients in the US and EU",
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