Long-term outcomes of enzyme replacement therapy from a large cohort of Korean patients with mucopolysaccharidosis IVA (Morquio A syndrome). [PDF]
Sung J +19 more
europepmc +1 more source
MUSCULOSKELETAL ALTERATIONS OF ORTHOPEDIC INTEREST IN MUCOPOLYSACCHARIDOSES. [PDF]
Matos MA, Lopes PS.
europepmc +1 more source
Advances in mucopolysaccharidosis research: the impact of mass spectrometry-based approaches. [PDF]
Ramarajan MG +3 more
europepmc +1 more source
Identification of Surrogate Biomarkers for Mucopolysaccharidosis Type IVA. [PDF]
Ago Y +4 more
europepmc +1 more source
Phenotype-Genotype Correlation in Morquio A Syndrome: Protocol for a Meta-Analysis.
Diaz-Ordoñez L +5 more
europepmc +1 more source
Outcomes of Lower Extremity Total Joint Arthroplasty in Patients With Skeletal Dysplasia: A Systematic Review. [PDF]
Guirguis P, Fowler L, Ricciardi BF.
europepmc +1 more source
Long-Term Liver-Targeted AAV8 Gene Therapy for Mucopolysaccharidosis IVA. [PDF]
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]
Mîndru DE +9 more
europepmc +1 more source
RNA-based therapeutic interventions for the management of Anderson-Fabry disease. [PDF]
Cagnin S.
europepmc +1 more source

