Rat models of musculoskeletal lysosomal storage disorders and their role in pre-clinical evaluation of gene therapy approaches. [PDF]
Marcó S, Muñoz S, Bosch F, Jimenez V.
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A Rare Case of Dyggve-Melchior-Clausen Syndrome: A Case Report. [PDF]
Chavan S +4 more
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Anesthetic and Airway Management in a Pediatric Patient with Morquio Syndrome: A Case Report. [PDF]
Garcia YKG, Reyes CRB.
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Mucopolysaccharidosis type IVA and severe hidradenitis suppurativa: A case series. [PDF]
Fialová J +3 more
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Collagen Type II-Targeting Lentiviral Gene Therapy for Mucopolysaccharidosis IVA. [PDF]
Celik B +4 more
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Exploring Multivalent Architectures for Binding and Stabilization of <i>N</i>-Acetylgalactosamine 6-Sulfatase. [PDF]
Davighi MG +8 more
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A Novel Missense Mutation of c.965C>T (p.Ala322Val) in the Human <i>GALNS</i> Gene Results in Severe Mucopolysaccharidosis Type IVA. [PDF]
Safavi M, Setoodeh A, Ghoddoosi M.
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Polysomnographic Analysis of Sleep-Disordered Breathing In Children With Mucopolysaccharidoses in Qatar: A Retrospective Study. [PDF]
Asir N, Al-Naimi AR.
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Mucopolysaccharidoses-What Clinicians Need to Know: A Clinical, Biochemical, and Molecular Overview. [PDF]
Lipiński P +11 more
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