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.
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.
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.
Alterations in Hurler-Scheie Syndrome Revealed by Mass Spectrometry-Based Proteomics and Phosphoproteomics Analysis.
Ramarajan MG, Parthasarathy KTS, Gaikwad KB, Joshi N, Garapati K, Kandasamy RK, Sharma J, Pandey A.
OMICS. 2024 Nov;28(11):548-562. doi: 10.1089/omi.2024.0171. Epub 2024 Oct 29.
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.
Mucopolysaccharidosis type I.
Wraith JE, Jones S.
Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:102-6.
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.
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.
Laronidase.
[No authors listed]
BioDrugs. 2002;16(4):316-8. doi: 10.2165/00063030-200216040-00009.
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.
