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The mutation in the MFSD8 (also known as CLN7 ) gene resulted in a truncated, and dysfunctional protein, and treatment with Milasen was able to effectively rescue seizure phenotypes and to increase neurological scores, temporarily improving quality of life of the patient [104]

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La administracin subcutnea suministra el pptido directamente en el espacio intersticial, donde puede acceder al sistema linftico y entrar a la circulacin con degradacin mnima

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