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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Single-Chain Variable Fragment Fusion Proteins for Targeted Delivery and Therapy. [PDF]
Pharmaceuticals (Basel)Yang L, Yin Y, Liu X, Guo B.
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AMCP Market Insights: The role of managed care in improving patient care in mucopolysaccharidoses. [PDF]
J Manag Care Spec PharmFlavin B +8 more
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[Research advances in pharmacotherapy for rare diseases in children]. [PDF]
Zhongguo Dang Dai Er Ke Za Zhi, 2023 Li JQ, Wang HJ.
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Editorial: Endo- and transcytotic pathways at the brain barriers. [PDF]
Front Drug DelivErickson MA, Nielsen MS.
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Advancing the Research and Development of Enzyme Replacement Therapies for Lysosomal Storage Diseases. [PDF]
GEN Biotechnol, 2022 Puhl AC, Ekins S.
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Early Recognition of Patients with Neuronopathic Forms of Mucopolysaccharidosis Type II in Clinical Practice. [PDF]
Med Sci (Basel)Buchinskaia NV +8 more
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Precision engineering of nanocarriers for transport across complex biological barriers. [PDF]
Int J Pharm XAcharya R +9 more
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Translational applicability of human blood-brain barrier spheroid models for the development of brain-penetrant therapeutic antibodies. [PDF]
Fluids Barriers CNSOhki S +8 more
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Nanoparticle-Mediated Targeted Drug Delivery Systems. [PDF]
PharmaceuticsTashima T, Tournier N.
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