Mucopolysaccharidosis Type II Screening, Diagnosis, and Management: A Literature Review and Practical Recommendations for Newborn Screening Programs and Health Care Providers to Support Families and Improve Outcomes. [PDF]
Int J Neonatal ScreenGaviglio A +7 more
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A Rare Case of Mucopolysaccharidosis Presenting With Dysostosis Multiplex and Preserved Intelligence in a Seven-Year-Old Girl From Northeast India. [PDF]
CureusRoy P, Roy P.
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A Contemporary Pathomechanistic Nosology of Inherited Lysosomal Disorders. [PDF]
J Inherit Metab DisMcCarron EP +7 more
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Dental Health in Pediatric Patients With Different Types of Mucopolysaccharidosis: Retrospective Cross-Sectional Study. [PDF]
JMIR Pediatr ParentWang M, Liu R, Xia T, Wang Y.
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Miglustat in Neuronopathic Lysosomal Storage Disorders: Biological Rationale, Clinical Evidence, and Limits of Repurposing. [PDF]
Int J Mol SciLipiński P, Węgrzyn G.
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Insights from the LysoNeo prospective cohort study to improve newborn screening of lysosomal diseases. [PDF]
Commun Med (Lond)Tebani A +18 more
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Mucopolysaccharidosis type VI in the Russian registry: Further insights and considerations. [PDF]
World J Clin PediatrMalik S.
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Role of the Gut-Liver-Kidney Axis in Disease Manifestation and Biomarker Alterations. [PDF]
Int J Mol SciMashima R, Ishige N.
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Impact of Mucopolysaccharidosis Type II in Young Children from the Caregiver's Perspective: A Qualitative Study. [PDF]
Adv TherBaldwin J +4 more
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