439. Prolonged Expression of Secreted Enzymes in Dogs After Liver Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Mucopolysaccharidoses Types I and VII [PDF]
Elena L. Aronovich +10 more
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Corneal Opacification in Infancy [PDF]
The purpose of this paper is to present a systematic classification of the more important conditions that can manifest as corneal opacification in early infancy and to state its differential diagnostic ...
Ching, Florencio C.
core +1 more source
Evaluating strategies to manage and endure challenging behaviors in mucopolysaccharidoses [PDF]
Nathan Grant
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The Biochemistry of the Mucopolysaccharidoses [PDF]
Gillian A. Blackham, D. N. Raine
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Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic [PDF]
Agata Fiumara +5 more
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MUSCULOSKELETAL ALTERATIONS OF ORTHOPEDIC INTEREST IN MUCOPOLYSACCHARIDOSES. [PDF]
Matos MA, Lopes PS.
europepmc +1 more source
Causal Therapies in Mucopolysaccharidoses: Enzyme Replacement Therapy [PDF]
Ara Vartanyan, Adriana M. Montaño
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Guidance for Home Enzyme Replacement Therapy in Children and Adolescents Diagnosed with Mucopolysaccharidoses: A Scoping Review Protocol. [PDF]
Oliveira VR +5 more
europepmc +1 more source
Molecular analysis of mucopolysaccharidoses: identification and characterization of pathogenic mutations in Indian population [PDF]
Anusha Uttarilli +9 more
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Orofacial manifestations in mucopolysaccharidoses: a comprehensive clinical and radiographic evaluation of 35 pediatric cases. [PDF]
Çoğulu D +5 more
europepmc +1 more source

