Glycosaminoglycan signatures in body fluids of mucopolysaccharidosis type II mouse model under long-term enzyme replacement therapy

[1] D’Avanzo F(2020)Mucopolysaccharidosis type II: one hundred years of research, diagnosis, and treatment Int J Mol Sci 21 E1258-307

[2] Rigon L(2017)Presentation and treatments for mucopolysaccharidosis type II (MPS II; Hunter syndrome) Expert Opin Orphan Drugs 5 295-1201

[3] Zanetti A(2018)Hematopoietic stem cell transplantation in mucopolysaccharidosis type II: a literature review and critical analysis J Inborn Errors Metab Screen 6 2326409818779097-75

[4] Tomanin R(2017)Treatment of mucopolysaccharidosis type II (Hunter syndrome): results from a systematic evidence review Genet Med Off J Am Coll Med Genet 19 1187-2275

[5] Stapleton M(2021)Enzyme replacement therapy with Pabinafusp alfa for Neuronopathic Mucopolysaccharidosis II: an integrated analysis of preclinical and clinical data Int J Mol Sci 22 10938-R129

[6] Kubaski F(2021)Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II Mol Ther Methods Clin Dev 21 67-118

[7] Mason RW(2018)FGF signaling deregulation is associated with early developmental skeletal defects in animal models for mucopolysaccharidosis type II (MPSII) Hum Mol Genet 27 2262-934

[8] Barth AL(2018)The pathogenesis of lysosomal storage disorders: beyond the engorgement of lysosomes to abnormal development and neuroinflammation Hum Mol Genet 27 R119-18

[9] Horovitz DDG(2020)Lysosomes as dynamic regulators of cell and organismal homeostasis Nat Rev Mol Cell Biol 21 101-1236

[10] Bradley LA(2007)The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome) J Inherit Metab Dis 30 924-1491

[1] D’Avanzo F(2020)Mucopolysaccharidosis type II: one hundred years of research, diagnosis, and treatment Int J Mol Sci 21 E1258-307

[2] Rigon L(2017)Presentation and treatments for mucopolysaccharidosis type II (MPS II; Hunter syndrome) Expert Opin Orphan Drugs 5 295-1201

[3] Zanetti A(2018)Hematopoietic stem cell transplantation in mucopolysaccharidosis type II: a literature review and critical analysis J Inborn Errors Metab Screen 6 2326409818779097-75

[4] Tomanin R(2017)Treatment of mucopolysaccharidosis type II (Hunter syndrome): results from a systematic evidence review Genet Med Off J Am Coll Med Genet 19 1187-2275

[5] Stapleton M(2021)Enzyme replacement therapy with Pabinafusp alfa for Neuronopathic Mucopolysaccharidosis II: an integrated analysis of preclinical and clinical data Int J Mol Sci 22 10938-R129

[6] Kubaski F(2021)Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II Mol Ther Methods Clin Dev 21 67-118

[7] Mason RW(2018)FGF signaling deregulation is associated with early developmental skeletal defects in animal models for mucopolysaccharidosis type II (MPSII) Hum Mol Genet 27 2262-934

[8] Barth AL(2018)The pathogenesis of lysosomal storage disorders: beyond the engorgement of lysosomes to abnormal development and neuroinflammation Hum Mol Genet 27 R119-18

[9] Horovitz DDG(2020)Lysosomes as dynamic regulators of cell and organismal homeostasis Nat Rev Mol Cell Biol 21 101-1236

[10] Bradley LA(2007)The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome) J Inherit Metab Dis 30 924-1491