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This is first clinical study using brain-penetrating insulin receptor antibodyiduronidase fusion proteins in the context of paediatric patients with severe Hurler syndrome (mucopolysaccharidosis type I)

Antiandrogenic effect of 16-substituted, non-substituted and D-homopregnane derivatives.Chem

Schedule: One subcutaneous injection a week across an 816 week course, with the dose raised in phases

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