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Conjugating TEM8-targeting antibodies with cytotoxic monomethyl auristatin E was successful in specifically directing the drug to the tumor microenvironment of orthotopic tumors and patient derived xenografts, significantly inducing regression or eradication of tumor growth in pre-clinical models [323]

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SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy

10 ng/kg, as retrobulbar application, 1 g