Effect of enzyme replacement therapy on the growth of patients with Morquio A
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Yasuyuki Suzuki | W. Mackenzie | Hironori Kobayashi | S. Yamaguchi | S. Tomatsu | R. Mason | Molly Stapleton | C. Doherty | Matthew Piechnik
[1] F. Deodato,et al. Enzyme replacement therapy: efficacy and limitations , 2018, Italian Journal of Pediatrics.
[2] S. Khan,et al. Glycosaminoglycans analysis in blood and urine of patients with mucopolysaccharidosis. , 2018, Molecular genetics and metabolism.
[3] C. Pizarro,et al. Molecular genetics and metabolism, special edition: Diagnosis, diagnosis and prognosis of Mucopolysaccharidosis IVA. , 2018, Molecular genetics and metabolism.
[4] Yasuyuki Suzuki,et al. Growth impairment in mucopolysaccharidoses. , 2018, Molecular genetics and metabolism.
[5] S. Tomatsu,et al. Neurophysiology of hearing in patients with mucopolysaccharidosis type IV. , 2018, Molecular genetics and metabolism.
[6] C. Pizarro,et al. Natural history of Morquio A patient with tracheal obstruction from birth to death , 2017, Molecular genetics and metabolism reports.
[7] Y. Puckett,et al. Enzyme Replacement Therapy with Elosulfase alfa for Mucopolysaccharidosis IVA (Morquio A Syndrome): Milestones and Challenges , 2017 .
[8] Marianne Rohrbach,et al. Epidemiology of mucopolysaccharidoses. , 2017, Molecular genetics and metabolism.
[9] S. Khan,et al. Mucopolysaccharidosis IVA and glycosaminoglycans. , 2017, Molecular genetics and metabolism.
[10] S. Ragot,et al. 30 months follow-up of an early enzyme replacement therapy in a severe Morquio A patient: About one case , 2016, Molecular genetics and metabolism reports.
[11] C. Pizarro,et al. Surgical Reconstruction for Severe Tracheal Obstruction in Morquio A Syndrome. , 2016, The Annals of thoracic surgery.
[12] T. Rahman,et al. Activity of daily living for Morquio A syndrome. , 2016, Molecular genetics and metabolism.
[13] Yasuyuki Suzuki,et al. Hematopoietic stem cell transplantation for Morquio A syndrome. , 2016, Molecular genetics and metabolism.
[14] C. Pizarro,et al. Obstructive airway in Morquio A syndrome, the past, the present and the future. , 2016, Molecular genetics and metabolism.
[15] T. Shimada,et al. Enzyme replacement therapy for treating mucopolysaccharidosis type IVA (Morquio A syndrome): effect and limitations , 2015, Expert opinion on orphan drugs.
[16] A. Shaywitz,et al. Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y , 2015, Pediatric Research.
[17] T. Shimada,et al. Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome , 2015, Drug design, development and therapy.
[18] C. Hendriksz,et al. Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial. , 2015, Molecular genetics and metabolism.
[19] W. Sly,et al. Therapies for the bone in mucopolysaccharidoses. , 2015, Molecular genetics and metabolism.
[20] A. Shaywitz,et al. Pharmacokinetic and Pharmacodynamic Evaluation of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients with Morquio A Syndrome , 2014, Clinical Pharmacokinetics.
[21] T. Fleming,et al. Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study , 2014, Journal of Inherited Metabolic Disease.
[22] Yasuyuki Suzuki,et al. Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA , 2014, Molecular genetics and metabolism reports.
[23] G. Mitchell,et al. Mucopolysaccharidosis IVA: correlation between genotype, phenotype and keratan sulfate levels. , 2013, Molecular genetics and metabolism.
[24] C. Hendriksz,et al. Review of clinical presentation and diagnosis of mucopolysaccharidosis IVA. , 2013, Molecular genetics and metabolism.
[25] R. Giugliani,et al. Impairment of Body Growth in Mucopolysaccharidoses , 2012 .
[26] J. Clarke,et al. Treatment of Lysosomal Storage Disorders , 2012, Drugs.
[27] Y. Suzuki,et al. Mucopolysaccharidosis type IVA (Morquio A disease): clinical review and current treatment. , 2011, Current pharmaceutical biotechnology.
[28] Hirotaka Oikawa,et al. Enhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.
[29] S. Tomatsu,et al. Sacral dimple: incidental findings from newborn evaluation (Case Presentation) , 2009, Acta paediatrica.
[30] W. Sly,et al. Acidic amino acid tag enhances response to enzyme replacement in mucopolysaccharidosis type VII mice. , 2008, Molecular genetics and metabolism.
[31] S. Tomatsu,et al. Growth charts for patients affected with Morquio A disease , 2008, American journal of medical genetics. Part A.
[32] W. Sly,et al. Enzyme replacement therapy in a murine model of Morquio A syndrome. , 2007, Human molecular genetics.
[33] L. de Meirleir,et al. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy , 2007, European Journal of Pediatrics.
[34] Avan Aihie Sayer,et al. Growth in early life predicts bone strength in late adulthood: The Hertfordshire Cohort Study , 2007, Bone.
[35] S. Tomatsu,et al. International Morquio A Registry: Clinical manifestation and natural course of Morquio A disease , 2007, Journal of Inherited Metabolic Disease.
[36] S. Tomatsu,et al. Determinant factors of spectrum of missense variants in mucopolysaccharidosis IVA gene. , 2006, Molecular genetics and metabolism.
[37] W. Sly,et al. Enhancement of drug delivery to bone: characterization of human tissue-nonspecific alkaline phosphatase tagged with an acidic oligopeptide. , 2006, Molecular genetics and metabolism.
[38] M. Connock,et al. A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1. , 2006, Health technology assessment.
[39] O. Amaral,et al. Prevalence of lysosomal storage diseases in Portugal , 2004, European Journal of Human Genetics.
[40] J. Belmont,et al. Enzyme-replacement therapy in mucopolysaccharidosis I. , 2001, The New England journal of medicine.
[41] Y. Masamune,et al. Novel Drug Delivery System to Bone Using Acidic Oligopeptide: Pharmacokinetic Characteristics and Pharmacological Potential , 2001, Journal of drug targeting.
[42] D. Applegarth,et al. Incidence of Inborn Errors of Metabolism in British Columbia, 1969–1996 , 2000, Pediatrics.
[43] R. A. Wevers,et al. The frequency of lysosomal storage diseases in The Netherlands , 1999, Human Genetics.
[44] P. Meikle,et al. Prevalence of lysosomal storage disorders. , 1999, JAMA.
[45] J. Nelson,et al. Incidence of the mucopolysaccharidoses in Northern Ireland , 1997, Human Genetics.