Current and Emerging Therapies for Mucopolysaccharidoses
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Publisher
Springer International Publishing
Link
http://link.springer.com/content/pdf/10.1007/164_2019_263
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1. Idursulfase Beta (Hunterase) Therapeutic Experience in a Patient with Mucopolysaccharidosis Type II;Current Pediatrics;2023-08-25
2. Alterations of Plasmatic Biomarkers of Neurodegeneration in Mucopolysaccharidosis Type II Patients Under Enzyme Replacement Therapy;Cell Biochemistry and Biophysics;2023-07-20
3. Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells;Human Gene Therapy;2022-12-01
4. Hunter Syndrome: The Phenotype of a Rare Storage Disease;Cureus;2022-02-07
5. Loss of Function of Mutant IDS Due to Endoplasmic Reticulum-Associated Degradation: New Therapeutic Opportunities for Mucopolysaccharidosis Type II;International Journal of Molecular Sciences;2021-11-12
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