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Kathryn L. Brammeier

University of Manchester

5

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30

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Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: Update on phase I/II clinical trial

2021 Molecular Genetics and Metabolism
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Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress - NCT04201405

2020 Blood
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Validation of ELISA and cellular uptake inhibition assays to detect and quantify antibodies raised against sebelipase alfa in lysosomal acid lipase deficiency

2020 Molecular Genetics and Metabolism
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The lysosomal storage disorders mucolipidosis type II, type III alpha/beta, and type III gamma: Update on GNPTAB and GNPTG mutations

2019 Human Mutation
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How many sulphatase deficiencies become multiple: An unusual presentation of multiple sulphatase deficiency

2018 Molecular Genetics and Metabolism
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