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Kiran C. Patki

Alexion Pharmaceuticals

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Final results of the phase 1/2, open-label clinical study of intravenous recombinant human N-acetyl-α-d-glucosaminidase (SBC-103) in children with mucopolysaccharidosis IIIB

2019 Molecular Genetics and Metabolism
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Enzymothérapie substitutive chez des patients atteints de mucopolysaccharidose iiib

2017
- Sandra Rojas-Caro
- Kiran C. Patki
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Novel NAGLU variants associated with MPS IIIB and in vitro analysis of residual enzymatic activities

2017 Molecular Genetics and Metabolism
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Evaluation of blood–brain barrier integrity and structural abnormalities in MPS IIIB patients using cerebrospinal fluid/serum albumin index (CSF-AI) and multimodal MRI

2015 Molecular Genetics and Metabolism
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