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mucopolysaccharidoses Ⅲ B相关文献:
Mucopolysaccharidosis VI.
Valayannopoulos V, Nicely H, Harmatz P, Turbeville S.
Orphanet J Rare Dis. 2010 Apr 12;5:5. doi: 10.1186/1750-1172-5-5.
PMID:20385007
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
Mucopolysaccharidosis VI: pathophysiology, diagnosis and treatment.
Harmatz P, Shediac R.
Front Biosci (Landmark Ed). 2017 Jan 1;22(3):385-406. doi: 10.2741/4490.
PMID:27814620
Anakinra in Sanfilippo syndrome: a phase 1/2 trial.
Polgreen LE, Chen AH, Pak Y, Luzzi A, Morales Garval A, Acevedo J, Bitan G, Iacovino M, O'Neill C, Eisengart JB.
Nat Med. 2024 Sep;30(9):2473-2479. doi: 10.1038/s41591-024-03079-3. Epub 2024 Jun 21.
PMID:38907160
Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA.
Giaccio M, Monaco A, Galiano L, Parente A, Borzacchiello L, Rubino R, Klärner FG, Killa D, Perna C, Piccolo P, Marotta M, Pan X, Khijniak M, Siddique I, Schrader T, Pshezhetsky AV, Sorrentino NC, Bitan G, Fraldi A.
Mol Ther. 2024 Nov 6;32(11):4108-4121. doi: 10.1016/j.ymthe.2024.09.030. Epub 2024 Sep 28.
PMID:39342429
Mucopolysaccharidoses-What Clinicians Need to Know: A Clinical, Biochemical, and Molecular Overview.
Lipiński P, Różdżyńska-Świątkowska A, Wiśniewska K, Rusecka J, Ługowska A, Żuber Z, Jezela-Stanek A, Cyske Z, Gaffke L, Pierzynowska K, Węgrzyn G, Tylki-Szymańska A.
Biomolecules. 2025 Oct 14;15(10):1448. doi: 10.3390/biom15101448.
PMID:41154677
Substrate reduction therapies for mucopolysaccharidoses.
Jakóbkiewicz-Banecka J, Piotrowska E, Gabig-Cimińska M, Borysiewicz E, Słomińska-Wojewódzka M, Narajczyk M, Węgrzyn A, Węgrzyn G.
Curr Pharm Biotechnol. 2011 Nov;12(11):1860-5. doi: 10.2174/138920111798376932.
PMID:21902626
Catabolic disorders of complex carbohydrates.
Spranger JW.
Postgrad Med J. 1977 Aug;53(622):441-9. doi: 10.1136/pgmj.53.622.441.
PMID:411121
Immunology of neonatal gene transfer.
Ponder KP.
Curr Gene Ther. 2007 Oct;7(5):403-10. doi: 10.2174/156652307782151434.
PMID:17979686
Development of a novel tool for individual treatment trials in mucopolysaccharidosis.
Wiesinger AM, Bigger B, Giugliani R, Lampe C, Scarpa M, Moser T, Kampmann C, Zimmermann G, Lagler FB.
J Inherit Metab Dis. 2025 Jan;48(1):e12816. doi: 10.1002/jimd.12816. Epub 2024 Nov 21.
PMID:39572375
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