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mucopolysaccharidoses Ⅲ D相关文献:
Glaucoma in mucopolysaccharidoses.
Kong W, Zhang J, Lu C, Ding Y, Meng Y.
Orphanet J Rare Dis. 2021 Jul 15;16(1):312. doi: 10.1186/s13023-021-01935-w.
PMID:34266471
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
Gentner B, Tucci F, Galimberti S, Fumagalli F, De Pellegrin M, Silvani P, Camesasca C, Pontesilli S, Darin S, Ciotti F, Sarzana M, Consiglieri G, Filisetti C, Forni G, Passerini L, Tomasoni D, Cesana D, Calabria A, Spinozzi G, Cicalese MP, Calbi V, Migliavacca M, Barzaghi F, Ferrua F, Gallo V, Miglietta S, Zonari E, Cheruku PS, Forni C, Facchini M, Corti A, Gabaldo M, Zancan S, Gasperini S, Rovelli A, Boelens JJ, Jones SA, Wynn R, Baldoli C, Montini E, Gregori S, Ciceri F, Valsecchi MG, la Marca G, Parini R, Naldini L, Aiuti A, Bernardo ME; MPSI Study Group.
N Engl J Med. 2021 Nov 18;385(21):1929-1940. doi: 10.1056/NEJMoa2106596.
PMID:34788506
Bow Hunter's Syndrome.
Regenhardt RW, Kozberg MG, Dmytriw AA, Vranic JE, Stapleton CJ, Silverman SB, Patel AB.
Stroke. 2022 Jan;53(1):e26-e29. doi: 10.1161/STROKEAHA.121.037253. Epub 2021 Dec 6.
PMID:34865507
Molecular characterization of 355 mucopolysaccharidosis patients reveals 104 novel mutations.
Pollard LM, Jones JR, Wood TC.
J Inherit Metab Dis. 2013 Mar;36(2):179-87. doi: 10.1007/s10545-012-9533-7. Epub 2012 Sep 14.
PMID:22976768
[Mucopolysaccharidoses: diagnosis and management].
Chabrol B, Cano A.
Arch Pediatr. 2014 Jun;21 Suppl 1:S1-3. doi: 10.1016/S0929-693X(14)72252-3.
PMID:25063378
Gene therapy for Mucopolysaccharidoses.
Sawamoto K, Chen HH, Alméciga-Díaz CJ, Mason RW, Tomatsu S.
Mol Genet Metab. 2018 Feb;123(2):59-68. doi: 10.1016/j.ymgme.2017.12.434. Epub 2017 Dec 26.
PMID:29295764
Idursulfase: I2S, iduronato-2-sulfatase.
[No authors listed]
Drugs R D. 2006;7(4):254-8. doi: 10.2165/00126839-200607040-00005.
PMID:16784250
Novel therapies for mucopolysaccharidosis type III.
Seker Yilmaz B, Davison J, Jones SA, Baruteau J.
J Inherit Metab Dis. 2021 Jan;44(1):129-147. doi: 10.1002/jimd.12316. Epub 2020 Sep 28.
PMID:32944950
The diagnosis and management of mucopolysaccharidosis type II.
Mao SJ, Chen QQ, Dai YL, Dong GP, Zou CC.
Ital J Pediatr. 2024 Oct 8;50(1):207. doi: 10.1186/s13052-024-01769-9.
PMID:39380047
Mucopolysaccharidosis IVA and glycosaminoglycans.
Khan S, Alméciga-Díaz CJ, Sawamoto K, Mackenzie WG, Theroux MC, Pizarro C, Mason RW, Orii T, Tomatsu S.
Mol Genet Metab. 2017 Jan-Feb;120(1-2):78-95. doi: 10.1016/j.ymgme.2016.11.007. Epub 2016 Nov 29.
PMID:27979613
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