Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB.

Furthermore, there was a highly significant, linear correlation (r = 0.95, P < 0.0001) between CSF HS-NRE concentration and liver weight for treated and untreated MPS IIIB dogs ( Fig. 4B ).

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The MPS IIIB dogs treated with 48 mg TA showed a trend toward improved performance over those treated with 12 mg ( Fig. 6A ); learning ability in the 48 mg dose group was similar to unaffected dogs and better than vehicle-treated MPS IIIB dogs.

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