Results 151 to 160 of about 4,093 (193)

Long-term outcomes of enzyme replacement therapy from a large cohort of Korean patients with mucopolysaccharidosis IVA (Morquio A syndrome). [PDF]

open access: yesMol Genet Metab Rep
Sung J   +19 more
europepmc   +1 more source

Phenotype-Genotype Correlation in Morquio A Syndrome: Protocol for a Meta-Analysis.

open access: yesJMIR Res Protoc
Diaz-Ordoñez L   +5 more
europepmc   +1 more source

Long-Term Liver-Targeted AAV8 Gene Therapy for Mucopolysaccharidosis IVA. [PDF]

open access: yesCurr Issues Mol Biol
Khan SA   +5 more
europepmc   +1 more source

Congenital Heart Malformations Masked by Infantile Gangliosidosis-Case Report and Growing Evidence for Metabolic Disease-Associated Aortopathies. [PDF]

open access: yesDiagnostics (Basel)
Mîndru DE   +9 more
europepmc   +1 more source

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