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Hurler-Scheie syndrome相关文献:
Heart valve disease in Hurler-Scheie syndrome.
García Del Rey MDC, Castrodeza J, Pinto Á, Espinosa Castro MÁ, Muñoz Delgado C, Fernández-Avilés F.
Cardiol J. 2022;29(5):875-877. doi: 10.5603/CJ.a2022.0066. Epub 2022 Jul 12.
PMID:35818796
Hurler-Scheie syndrome in Niger: a case series.
Assadeck H, Toudou Daouda M, Bako H, Hassane Djibo F.
J Med Case Rep. 2019 Apr 25;13(1):102. doi: 10.1186/s13256-019-2047-2.
PMID:31018863
Scleral Histopathologic Findings of Hurler-Scheie Syndrome With Refractory Glaucoma.
Dolezal KA, Jacobson A, Besirli CG, Elner V, Bohnsack BL.
JAMA Ophthalmol. 2022 Mar 1;140(3):285-287. doi: 10.1001/jamaophthalmol.2021.5806.
PMID:35050321
Musculoskeletal complications associated with lysosomal storage disorders: Gaucher disease and Hurler-Scheie syndrome (mucopolysaccharidosis type I).
Pastores GM, Meere PA.
Curr Opin Rheumatol. 2005 Jan;17(1):70-8. doi: 10.1097/01.bor.0000147283.40529.13.
PMID:15604908
Mucopolysaccharidosis type I.
Wraith JE, Jones S.
Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:102-6.
PMID:25345091
The Beginning of the End of Allogeneic Transplantation for Hurler Syndrome?
Kharbanda S, Dvorak CC.
N Engl J Med. 2021 Nov 18;385(21):2003-2004. doi: 10.1056/NEJMe2116020.
PMID:34788512
Early disease progression of Hurler syndrome.
Kiely BT, Kohler JL, Coletti HY, Poe MD, Escolar ML.
Orphanet J Rare Dis. 2017 Feb 14;12(1):32. doi: 10.1186/s13023-017-0583-7.
PMID:28193245
Laronidase.
[No authors listed]
BioDrugs. 2002;16(4):316-8. doi: 10.2165/00063030-200216040-00009.
PMID:12196045
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
Consiglieri G, Tucci F, De Pellegrin M, Guerrini B, Cattoni A, Risca G, Scarparo S, Sarzana M, Pontesilli S, Mellone R, Gasperini S, Galimberti S, Silvani P, Filisetti C, Darin S, Forni G, Miglietta S, Santi L, Facchini M, Corti A, Fumagalli F, Cicalese MP, Calbi V, Migliavacca M, Barzaghi F, Ferrua F, Gallo V, Recupero S, Canarutto D, Doglio M, Tedesco L, Volpi N, Rovelli A, la Marca G, Valsecchi MG, Zancan S, Ciceri F, Naldini L, Baldoli C, Parini R, Gentner B, Aiuti A, Bernardo ME.
Sci Transl Med. 2024 May;16(745):eadi8214. doi: 10.1126/scitranslmed.adi8214. Epub 2024 May 1.
PMID:38691622
Hurler syndrome: past, present, and future.
Peters C, Shapiro EG, Krivit W.
J Pediatr. 1998 Jul;133(1):7-9. doi: 10.1016/s0022-3476(98)70170-2.
PMID:9672503
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