Therapy-type related long-term outcomes in mucopolysaccaridosis type II (Hunter syndrome) - Case series.

Zerjav Tansek, Mojca; Kodric, Jana; Klemencic, Simona; Boelens, Jaap Jan; van Hasselt, Peter M; Drole Torkar, Ana; Doric, Maja; Koren, Alenka; Avcin, Simona; Battelino, Tadej; Groselj, Urh

Zerjav Tansek, Mojca; Kodric, Jana; Klemencic, Simona; Boelens, Jaap Jan; van Hasselt, Peter M; Drole Torkar, Ana; Doric, Maja; Koren, Alenka; Avcin, Simona; Battelino, Tadej; Groselj, Urh.

Afiliación

Zerjav Tansek M; Department of Endocrinology, Diabetes, and Metabolic Diseases, University Children's Hospital, UMC Ljubljana, Ljubljana, Slovenia.
Kodric J; Faculty of Medicine, University of Ljubljana, Ljubljana, Slovenia.
Klemencic S; Department of Child Psychiatry, University Children's Hospital, UMC Ljubljana, Ljubljana, Slovenia.
Boelens JJ; Department of Endocrinology, Diabetes, and Metabolic Diseases, University Children's Hospital, UMC Ljubljana, Ljubljana, Slovenia.
van Hasselt PM; Department of Pediatric Immunology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht, the Netherlands.
Drole Torkar A; Stem Cell Transplantation and Cellular Therapies Program, Department Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Doric M; Department of Metabolic Diseases, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands.
Koren A; Department of Endocrinology, Diabetes, and Metabolic Diseases, University Children's Hospital, UMC Ljubljana, Ljubljana, Slovenia.
Avcin S; Faculty of Medicine, University of Ljubljana, Ljubljana, Slovenia.
Battelino T; Department of Endocrinology, Diabetes, and Metabolic Diseases, University Children's Hospital, UMC Ljubljana, Ljubljana, Slovenia.
Groselj U; Department of Endocrinology, Diabetes, and Metabolic Diseases, University Children's Hospital, UMC Ljubljana, Ljubljana, Slovenia.

Mol Genet Metab Rep ; 28: 100779, 2021 Sep.

Article en En | MEDLINE | ID: mdl-34258227

Palabras clave

CNS, central nervous system; ENT, ear nose and throat specialist; ERT, enzyme replacement therapy; GAG, glycosaminoglycans; HSCT, enzyme replacement therapy; HSCT, hematopoietic stem cell transplantation; Hematopoietic stem cell transplantation; Hunter syndrome; I2S, iduronate-2-sulfatase; LSD, lysosomal storage disease; MPS; MPS, mucopolysaccharidosis; Mucopolysaccharidosis type II

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Mol Genet Metab Rep Año: 2021 Tipo del documento: Article País de afiliación: Eslovenia Pais de publicación: Estados Unidos

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