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A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis.

Kell P, Sidhu R, Qian M, Mishra S, Nicoli ER, D'Souza P, Tifft CJ, Gross AL, Gray-Edwards HL, Martin DR, Sena-Esteves M, Dietzen DJ, Singh M, Luo J, Schaffer JE, Ory DS, Jiang X. A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis. EBioMedicine. 2023 Jun; 92:104627.

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