Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa [PDF]
Orphanet Journal of Rare Diseases, 2017 Background This post hoc subanalysis examined outcomes in adult patients with Morquio A (mucopolysaccharidosis IVA) who received enzyme replacement therapy (ERT) with elosulfase alfa over a 120-weeks period. Patients ≥18 years of age evaluated in an open-
D. Hughes +8 more
doaj +10 more sources
Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England [PDF]
Orphanet Journal of Rare Diseases, 2021 Background We present baseline characteristics and follow-up data of a Managed Access Agreement (MAA), including patients with mucopolysaccharidosis IVA (MPS IVA) receiving elosulfase alfa enzyme replacement therapy (ERT) in England on a conditional ...
Maureen Cleary +11 more
doaj +4 more sources
Clinical Utility of Elosulfase Alfa in the Treatment of Morquio A Syndrome [PDF]
Drug Design, Development and Therapy, 2022 Chung-Lin Lee,1– 5 Chih-Kuang Chuang,6,7 Huei-Ching Chiu,1 Ru-Yi Tu,6 Yun-Ting Lo,5 Ya-Hui Chang,1,5 Shuan-Pei Lin,1,3,5,6,8 Hsiang-Yu Lin1,3– 6,9 1Department of Pediatrics, MacKay Memorial Hospital, Taipei, Taiwan; 2Institute of Clinical Medicine ...
Lee CL +7 more
doaj +5 more sources
Enzyme-Loaded Gel Core Nanostructured Lipid Carriers to Improve Treatment of Lysosomal Storage Diseases: Formulation and In Vitro Cellular Studies of Elosulfase Alfa-Loaded Systems [PDF]
Pharmaceutics, 2019 Mucopolysaccharidosis IVA (Morquio A) is a rare inherited metabolic disease caused by deficiency of the lysosomal enzyme N-acetylgalatosamine-6-sulfate-sulfatase (GALNS). Until now, treatments employed included hematopoietic stem cell transplantation and
J. Víctor Álvarez +9 more
doaj +5 more sources
Elosulfase alfa in the treatment of mucopolysaccharidosis type IVA: insights from the first managed access agreement [PDF]
Orphanet Journal of Rare Diseases, 2021 Managed access agreements provide a crucial mechanism whereby real-world data can be collected systematically to reduce uncertainty around available clinical and economic data, whilst providing the opportunity to identify patient sub-populations who are ...
Bob Stevens +5 more
doaj +5 more sources
Clinical characteristics and effects of enzyme replacement therapy with elosulfase alfa in Korean patients with mucopolysaccharidosis type IVA [PDF]
Molecular Genetics and Metabolism Reports, 2022 Mucopolysaccharidosis type IVA (MPS IVA) is a rare autosomal recessive disorder caused by a deficiency in N-acetylgalactosamine-6-sulfatase, which results in skeletal and connective tissue abnormalities, as well as various non-skeletal manifestations ...
Seung Hoon Lee +4 more
doaj +4 more sources
Elosulfase alfa enzyme replacement therapy attenuates disease progression in a non-ambulatory Japanese patient with Morquio A syndrome (case report) [PDF]
Molecular Genetics and Metabolism Reports, 2017 Enzyme replacement therapy (ERT) with elosulfase alfa is the only approved therapy in Japan for patients with Morquio A syndrome, a lysosomal storage disorder inherited in an autosomal recessive fashion.
Misako Hiramatsu, Kimitoshi Nakamura
doaj +4 more sources
Elosulfase alfa for mucopolysaccharidosis type IVA: Real-world experience in 7 patients from the Spanish Morquio-A early access program [PDF]
Molecular Genetics and Metabolism Reports, 2018 There is a growing interest in evaluating the effectiveness of enzyme replacement therapy (ERT) with elosulfase alfa in patients with mucopolysaccharidosis type IVA (MPS-IVA) under real-world conditions.
Guillem Pintos-Morell +6 more
doaj +7 more sources
Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance [PDF]
Orphanet Journal of Rare Diseases, 2019 Introduction Mucopolysaccharidosis (MPS) IVA or Morquio A syndrome is an autosomal recessive lysosomal storage disorder (LSD) caused by deficiency of the N-acetylgalactosamine-6-sulfatase (GALNS) enzyme, which impairs lysosomal degradation of keratan ...
Mehmet Umut Akyol +30 more
doaj +9 more sources
Clinical features and health-related quality of life in adult patients with mucopolysaccharidosis IVA: the Spanish experience [PDF]
Orphanet Journal of Rare Diseases, 2021 Background Mucopolysaccharidosis (MPS) IVA or Morquio A syndrome is a progressive and disabling disease characterized by a deficiency of the enzyme N-acetylgalactosamine-6-sulphate sulphatase.
Pilar Quijada-Fraile +15 more
doaj +5 more sources