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The regular dosage form: Lyophilized Powder for Injection, Tablets,Capsules, Eye drops etc

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No data are available in the clinical setting

( Mutation detection and gene fusion analysis were performed using the customized panels QIASeq targeted DNA Panel for Solid Tumors (Qiagen) and QIAseq targeted RNAscan Panel (Qiagen)

1.How often do we need to come in for clinic visits and blood work (carnitine levels, acylcarnitine profile)