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Successful Elosulfase Alfa Desensitization Protocol in a Patient With Morquio A Syndrome
Pediatrics, 2022Patients with lysosomal storage diseases may require modifications to standard drug desensitization protocols; personalized medicine as well as development of new treatment options are needed.
Verónica C, Díaz Vidal +3 more
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Elosulfase alfa: First Global Approval
Drugs, 2014Elosulfase alfa (Vimizim™) is a recombinant form of N-acetylgalactosamine-6-sulfatase (GALNS) that was developed by BioMarin Pharmaceutical Inc. as an enzyme replacement therapy for patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome.
Mark, Sanford, Jin Han, Lo
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Drugs of Today, 2014
Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, is an inherited, lysosomal storage disorder caused by genetic mutations in N-acetylgalactosamine-6-sulfatase (GALNS) enzyme gene. GALNS is essential for breakdown of glycosaminoglycans.
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Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, is an inherited, lysosomal storage disorder caused by genetic mutations in N-acetylgalactosamine-6-sulfatase (GALNS) enzyme gene. GALNS is essential for breakdown of glycosaminoglycans.
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Elosulfase alfa for the treatment of mucopolysacchoridosis IVA
Expert Review of Endocrinology & Metabolism, 2015Mucopolysacchariodosis IVA is a lysosomal storage disorder characterized by deficiency of the enzyme N-acetylgalactosamine-6-sulfatase leading to accumulation of glycosaminoglycans. Mucopolysacchariodosis IVA affects many organs, especially the skeletal system.
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Expert Opinion on Orphan Drugs, 2017
ABSTRACTIntroduction: Mucopolysaccharidosis IVA (Morquio A syndrome) is an inherited, autosomal recessive disease caused by a deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS), which results in excessive lysosomal storage of keratan sulfate and chondroitin-6-sulfate in many tissues and organs.
Yana Puckett +2 more
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ABSTRACTIntroduction: Mucopolysaccharidosis IVA (Morquio A syndrome) is an inherited, autosomal recessive disease caused by a deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS), which results in excessive lysosomal storage of keratan sulfate and chondroitin-6-sulfate in many tissues and organs.
Yana Puckett +2 more
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BioDrugs, 2014
Elosulfase alfa (Vimizim(®)) is a recombinant form of the human lysosomal enzyme N-acetylgalactosamine-6-sulfatase (GALNS) that is lacking in patients with mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). It is the first, and currently only, disease-specific treatment option for this very rare, progressively degenerative, autosomal ...
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Elosulfase alfa (Vimizim(®)) is a recombinant form of the human lysosomal enzyme N-acetylgalactosamine-6-sulfatase (GALNS) that is lacking in patients with mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). It is the first, and currently only, disease-specific treatment option for this very rare, progressively degenerative, autosomal ...
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Shortened desensitization leading to a 2-year enzyme replacement therapy with elosulfase alfa
Annals of Allergy, Asthma & Immunology, 2021Hayrunnisa Bekis Bozkurt +3 more
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The Journal of Allergy and Clinical Immunology: In Practice, 2017
Hakan Guvenir +6 more
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Hakan Guvenir +6 more
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