PubMed for id: 7734

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38462612,38454486,38186847,38085235,38053935,37974184,37915038,37860991,37506513,37427450,37359415,36982718,36168594,36027721,35961250,35662814,35620452,35588317,34863240,34837645,34717704,34681597,34424960,33622387,33458989,33138761,33101985,32342708,31383595,29978271,29863901,28918469,28894675,28673849,28649514,28543354,28243577,27349225,25902842,25834948,25712379,24515576,23988379,23512580,23497636,23430900,23305961,22190500,21910981,21866356,21672014,21192662,20203116,20098308,19773189,19707363,18245410,18201153,17569950,17185020,17082248,16912829,16912578,16495038,8837099,8685031,7981716,6770331,6432613,3938449,2516792,2507197,2122274,1906048,1352274,228523,187158,150330,132078,112128,36945845,33781915,32773276,32751752,31877959,30595526,29884617,29633336,29606503,27837218,27331006,23726270,22666927,22286622,22279584,22105882,21528770,21319344,20509947,19697114,18580692,18174963,17876721,17459751,17206939,17091340,16699754,9712538,8723110,7858175,6410119,3931669,2118851,35768874,31536183,29843745,28256515,28211988,27246110,25110468,24917356,17264973,16962210,34070997,33508431,29618310,38605499,38545863,38542525,38332678,38310355,38241811,38026328,38013546,37922836,37797682,37792110,37577929,37470932,37357221,37340133,37286508,37188686,37130633,37023503,37014547,36907694,36840680,36807032,36541357,36445366,36331899,36329518,36260294,36257379,36226412,36107175,36077388,36029429,35929937,35916809,35882106,35835952,35816218,35810416,35773763,35759972,35568060,35563245,35563175,35537249,35536351,35331284,35226799,35226042,35152775,35144014,35125232,34990619,34980815,34893157,34830113,34813777,34797484,34794030,34725353,34670126,34622797,34483194,34480399,34360653,34289859,34251974,34193122,34173004,34170006,34031351,33960103,33741271,33676511,33674293,33402755,33389200,33382737,33344633,33330992,33290290,33210402,33038326,33004112,32935418,32927819,32741966,32707880,32631737,32623553,32619248,32563631,32522486,32508845,32502916,32448126,32312280,32188102,32070051,32043138,32009470,31941077,31926052,31905715,31895584,31834462,31757021,31639024,31544538,31477379,31469979,31341247,31225628,31140601,31071499,31046699,31039347,31029429,31022913,31005860,30994022,30953652,30639582,30566525,30499826,30443672,30409495,30409228,30375140,30261398,30249478,30213032,30143438,30064964,30042467,30024503,29866148,29860540,29801985,29801497,29779903,29718288,29648648,29580682,29506582,29314214,29289480,29210605,29210515,29168930,29168031,29073295,28974237,28887757,28860717,28820625,28640238,28610913,28595941,28593992,28513549,28501294,28478695,28477385,28464912,28458178,28334757,28207863,28186595,28171221,28095876,28077157,28011272,27999126,27986197,27916847,27913904,27888081,27883178,27873002,27829684,27789401,27789399,27789398,27789394,27764180,27724940,27699273,27695081,27590924,27512952,27510804,27491212,27251652,27228099,27187040,27169704,27146977,27112191,27016302,26968075,26962552,26897145,26846156,26845288,26768041,26762690,26633932,26592960,26588497,26520066,26479407,26360015,26297739,26297117,26051019,26046872,25976201,25952974,25922936,25887606,25851683,25836678,25797591,25761450,25721852,25690978,25622168,25572378,25547887,25541100,25503568,25468650,25468646,25458519,25410665,25402250,25395377,25391186,25345092,25344850,25231261,25219292,25169630,25141890,25096868,25044788,25038527,24979053,24908534,24836711,24806354,24781369,24780617,24705807,24596019,24550654,24510650,24454794,24423630,24399699,24384963,24368158,24277967,24268528,24238892,24234586,24202085,24201805,24190099,24125893,24100247,24083598,24056375,24011228,24005894,23954699,23867855,23844659,23837440,23815875,23800320,23726952,23707223,23691848,23690022,23621439,23537841,23529876,23468122,23404265,23401495,23375472,23307460,23232253,23221651,23084433,23022072,22999151,22960538,22929184,22926198,22847837,22704483,22622771,22610466,22492741,22433425,22414067,22383073,22381044,22246721,22227323,22194384,22153556,22077147,22071845,22059643,22037758,21995841,21983729,21834048,21829674,21824567,21800666,21732093,21706504,21605424,21593745,21567931,21530981,21529823,21524741,21518713,21465231,21447345,21439875,21427013,21420339,21334454,21327479,21319059,21291454,21235446,21233716,21211680,21204210,21150784,21108396,21062272,21055381,21045710,20978944,20973983,20950410,20934363,20886296,20876612,20709629,20652491,20638311,20503319,20382108,20354449,20136838,20104590,20052546,20040319,20040314,19933090,19901005,19860233,19821143,19761845[uid]

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Results by year

Year	Number of Results
1976	2
1977	1
1978	1
1979	1
1980	1
1983	1
1984	1
1985	2
1989	2
1990	2
1991	1
1992	1
1993	1
1994	2
1996	3
1998	1
2000	1
2006	8
2007	9
2008	4
2009	8
2010	26
2011	48
2012	32
2013	43
2014	45
2015	32
2016	45
2017	40
2018	37
2019	27
2020	34
2021	36
2022	41
2023	35
2024	15

500 results

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Page 1

Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II.

Yee KS, Lewis S, Evans E, Romano C, Alexanderian D. Yee KS, et al. Orphanet J Rare Dis. 2024 Mar 10;19(1):110. doi: 10.1186/s13023-024-03034-y. Orphanet J Rare Dis. 2024. PMID: 38462612 Free PMC article. Clinical Trial.

Analysis of caregiver perspectives on patients with mucopolysaccharidosis II treated with pabinafusp alfa: results of qualitative interviews in Japan.

Nakamura K, Sakai N, Hossain MA, Eisengart JB, Yamamoto T, Tanizawa K, So S, Schmidt M, Sato Y. Nakamura K, et al. Orphanet J Rare Dis. 2024 Mar 7;19(1):104. doi: 10.1186/s13023-024-03112-1. Orphanet J Rare Dis. 2024. PMID: 38454486 Free PMC article.

Characterization of orthopedic manifestations in patients with mucopolysaccharidosis II using data from 15 years of the Hunter Outcome Survey.

Link B, Botha J, Giugliani R. Link B, et al. JIMD Rep. 2023 Nov 27;65(1):17-24. doi: 10.1002/jmd2.12401. eCollection 2024 Jan. JIMD Rep. 2023. PMID: 38186847 Free PMC article.

Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage.

Catalano F, Vlaar EC, Dammou Z, Katsavelis D, Huizer TF, Zundo G, Hoogeveen-Westerveld M, Oussoren E, van den Hout HJMP, Schaaf G, Pike-Overzet K, Staal FJT, van der Ploeg AT, Pijnappel WWMP. Catalano F, et al. Hum Gene Ther. 2024 Apr;35(7-8):256-268. doi: 10.1089/hum.2023.177. Epub 2024 Feb 2. Hum Gene Ther. 2024. PMID: 38085235 Free PMC article.

Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey.

Lau H, Harmatz P, Botha J, Audi J, Link B. Lau H, et al. Mol Genet Metab Rep. 2023 Sep 8;37:101005. doi: 10.1016/j.ymgmr.2023.101005. eCollection 2023 Dec. Mol Genet Metab Rep. 2023. PMID: 38053935 Free PMC article.

Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study.

Muenzer J, Burton BK, Amartino HM, Harmatz PR, Gutiérrez-Solana LG, Ruiz-Garcia M, Wu Y, Merberg D, Alexanderian D, Jones SA. Muenzer J, et al. Orphanet J Rare Dis. 2023 Nov 16;18(1):357. doi: 10.1186/s13023-023-02805-3. Orphanet J Rare Dis. 2023. PMID: 37974184 Free PMC article.

A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT.

Yee KS, Chirila C, Davenport E, Mladsi D, Barnett C, Kronenberger WG. Yee KS, et al. Orphanet J Rare Dis. 2023 Nov 2;18(1):343. doi: 10.1186/s13023-023-02957-2. Orphanet J Rare Dis. 2023. PMID: 37915038 Free PMC article. Clinical Trial.

Transferrin Receptor-Targeted Iduronate-2-sulfatase Penetrates the Blood-Retinal Barrier and Improves Retinopathy in Mucopolysaccharidosis II Mice.

Imakiire A, Morimoto H, Suzuki H, Masuda T, Yoden E, Inoue A, Morioka H, Konaka T, Mori A, Shirasaka R, Kato R, Hirato T, Sonoda H, Minami K. Imakiire A, et al. Mol Pharm. 2023 Nov 6;20(11):5901-5909. doi: 10.1021/acs.molpharmaceut.3c00736. Epub 2023 Oct 20. Mol Pharm. 2023. PMID: 37860991 Free PMC article.

Cognitive and adaptive behaviors associated with disease severity and genotype in patients with mucopolysaccharidosis II.

Yee KS, Alexanderian D, Merberg D, Natarajan M, Wang S, Wu Y, Whiteman DAH. Yee KS, et al. Mol Genet Metab. 2023 Nov;140(3):107652. doi: 10.1016/j.ymgme.2023.107652. Epub 2023 Jul 13. Mol Genet Metab. 2023. PMID: 37506513 Free article.

Establishment of the Effectiveness of Early Versus Late Stem Cell Gene Therapy in Mucopolysaccharidosis II for Treating Central Versus Peripheral Disease.

Mandolfo O, Liao A, Singh E, O'leary C, Holley RJ, Bigger BW. Mandolfo O, et al. Hum Gene Ther. 2024 Apr;35(7-8):243-255. doi: 10.1089/hum.2023.002. Epub 2023 Aug 30. Hum Gene Ther. 2024. PMID: 37427450

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