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similar Morquio' s syndrome相关文献:
Fusions at the craniovertebral junction.
Ahmed R, Traynelis VC, Menezes AH.
Childs Nerv Syst. 2008 Oct;24(10):1209-24. doi: 10.1007/s00381-008-0607-7. Epub 2008 Apr 4.
PMID:18389260
Activity of daily living for Morquio A syndrome.
Yasuda E, Suzuki Y, Shimada T, Sawamoto K, Mackenzie WG, Theroux MC, Pizarro C, Xie L, Miller F, Rahman T, Kecskemethy HH, Nagao K, Morlet T, Shaffer TH, Chinen Y, Yabe H, Tanaka A, Shintaku H, Orii KE, Orii KO, Mason RW, Montaño AM, Fukao T, Orii T, Tomatsu S.
Mol Genet Metab. 2016 Jun;118(2):111-22. doi: 10.1016/j.ymgme.2016.04.005. Epub 2016 Apr 25.
PMID:27161890
Hurler's, Hunter's and Morquio's syndromes. A biochemical study in the light of current views of the underlying defects.
Dean MF, Muir H, Ewins RJ.
Biochem J. 1971 Aug;123(5):883-94. doi: 10.1042/bj1230883.
PMID:4256532
Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa.
Hughes D, Giugliani R, Guffon N, Jones SA, Mengel KE, Parini R, Matousek R, Hawley SM, Quartel A.
Orphanet J Rare Dis. 2017 May 23;12(1):98. doi: 10.1186/s13023-017-0634-0.
PMID:28535791
Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial.
Hendriksz CJ, Giugliani R, Harmatz P, Mengel E, Guffon N, Valayannopoulos V, Parini R, Hughes D, Pastores GM, Lau HA, Al-Sayed MD, Raiman J; STRIVE Investigators; Yang K, Mealiffe M, Haller C.
Mol Genet Metab. 2015 Feb;114(2):178-85. doi: 10.1016/j.ymgme.2014.08.012. Epub 2014 Sep 6.
PMID:25284089
Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.
Tomatsu S, Sawamoto K, Alméciga-Díaz CJ, Shimada T, Bober MB, Chinen Y, Yabe H, Montaño AM, Giugliani R, Kubaski F, Yasuda E, Rodríguez-López A, Espejo-Mojica AJ, Sánchez OF, Mason RW, Barrera LA, Mackenzie WG, Orii T.
Drug Des Devel Ther. 2015 Apr 1;9:1937-53. doi: 10.2147/DDDT.S68562. eCollection 2015.
PMID:25897204
Diagnosis of Morquio Syndrome in Dried Blood Spots Based on a New MRM-MS Assay.
Cozma C, Eichler S, Wittmann G, Flores Bonet A, Kramp GJ, Giese AK, Rolfs A.
PLoS One. 2015 Jul 6;10(7):e0131228. doi: 10.1371/journal.pone.0131228. eCollection 2015.
PMID:26147980
The Morquio syndrome: neuropathology and biochemistry.
Koto A, Horwitz AL, Suzuki K, Tiffany CW, Suzuki K.
Ann Neurol. 1978 Jul;4(1):26-36. doi: 10.1002/ana.410040106.
PMID:100045
Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study.
Long B, Tompkins T, Decker C, Jesaitis L, Khan S, Slasor P, Harmatz P, O'Neill CA, Schweighardt B.
Clin Ther. 2017 Jan;39(1):118-129.e3. doi: 10.1016/j.clinthera.2016.11.017. Epub 2016 Dec 10.
PMID:27955919
Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome.
Hendriksz CJ, Parini R, AlSayed MD, Raiman J, Giugliani R, Solano Villarreal ML, Mitchell JJ, Burton BK, Guelbert N, Stewart F, Hughes DA, Berger KI, Slasor P, Matousek R, Jurecki E, Shaywitz AJ, Harmatz PR.
Mol Genet Metab. 2016 Sep;119(1-2):131-43. doi: 10.1016/j.ymgme.2016.05.018. Epub 2016 Jun 16.
PMID:27380995
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