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Academic Journal

Long term follow-up after haematopoietic stem cell transplantation for mucopolysaccharidosis type I-H: a retrospective study of 51 patients

Subjects: Neurological disorders; Paediatrics; [SDV]Life Sciences [q-bio]

  • Source: ISSN: 0268-3369 ; Bone Marrow Transplantation ; https://u-paris.hal.science/hal-04094846 ; Bone Marrow Transplantation, 2022, 58, pp.295 - 302. ⟨10.1038/s41409-022-01886-1⟩.

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Academic Journal

Gene therapies in pediatric ophthalmology

Subjects: gene therapy; children; voretigene neparvovec

  • Source: ISSN: 2674-0826 ; Frontiers in Ophthalmology ; https://hal.science/hal-04544311 ; Frontiers in Ophthalmology, 2023, 3, pp.1188522. ⟨10.3389/fopht.2023.1188522⟩.

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