Clinical and molecular spectrum of mucopolysaccharidosis IVA in Iraqi children: Allele-specific genotype-phenotype trends and novel GALNS variants. [PDF]
Mol Genet Metab RepWahhab SBA, Thejeal RF.
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Long-Term Liver-Targeted AAV8 Gene Therapy for Mucopolysaccharidosis IVA. [PDF]
Curr Issues Mol BiolKhan SA +5 more
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Three-dimensional human mucopolysaccharidosis IVA chondrocyte culture reveals significant impairments in the lysosomal-mitochondrial crosstalk. [PDF]
Sci RepLeal AF, Saikia S, Khan SA, Tomatsu S.
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Craniovertebral Junction Compression in Patients With Morquio Syndrome: Case Series and Literature Review. [PDF]
CureusRakshit P +4 more
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Mucopolysaccharidoses: A biochemical study under limited resources. [PDF]
Mol Genet Metab RepBouzid F +7 more
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Recent advances in mucopolysaccharidosis IVA treatment. [PDF]
Orphanet J Rare DisLeal AF, Pachajoa H.
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A Rare Compound Heterozygous NAGLU Gene Mutation in Two Siblings with Mucopolysaccharidosis type Iiib. [PDF]
Iran J PatholVahedi-Larijani L +5 more
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Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS). [PDF]
Orphanet J Rare DisStepien KM +14 more
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Advances in mucopolysaccharidosis research: the impact of mass spectrometry-based approaches. [PDF]
Clin ProteomicsRamarajan MG +3 more
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