Cargando…

Clinical Utility of Elosulfase Alfa in the Treatment of Morquio A Syndrome

Mucopolysaccharidosis type IVA (MPS IVA or Morquio A) is an autosomal recessive disorder and is one of the lysosomal storage diseases. Patients with MPS IVA have a striking skeletal phenotype but normal intellect. The phenotypic continuum of MPS IVA ranges from severe and rapid progress to mild and...

Descripción completa

Detalles Bibliográficos
Autores principales:	Lee, Chung-Lin, Chuang, Chih-Kuang, Chiu, Huei-Ching, Tu, Ru-Yi, Lo, Yun-Ting, Chang, Ya-Hui, Lin, Shuan-Pei, Lin, Hsiang-Yu
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Dove 2022
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8759989/ https://www.ncbi.nlm.nih.gov/pubmed/35046639 http://dx.doi.org/10.2147/DDDT.S219433

Ejemplares similares

Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis
por: Lee, Chung-Lin, et al.
Publicado: (2022)

Pharmacokinetic and Pharmacodynamic Evaluation of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients with Morquio A Syndrome
por: Qi, Yulan, et al.
Publicado: (2014)

Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome
por: Hendriksz, Christian J., et al.
Publicado: (2016)

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
por: Hendriksz, Christian J., et al.
Publicado: (2014)

Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y
por: Jones, Simon A., et al.
Publicado: (2015)

Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open‐label, phase 2 study
por: Harmatz, Paul R., et al.
Publicado: (2016)

Role of elosulfase alfa in mucopolysaccharidosis IVA
por: Regier, Debra S, et al.
Publicado: (2016)

Elosulfase alfa for mucopolysaccharidosis type IVA: Real-world experience in 7 patients from the Spanish Morquio-A early access program
por: Pintos-Morell, Guillem, et al.
Publicado: (2018)

Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa
por: Hughes, D., et al.
Publicado: (2017)

Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double‐blind, pilot study
por: Burton, Barbara K., et al.
Publicado: (2015)

Elosulfase alfa enzyme replacement therapy attenuates disease progression in a non-ambulatory Japanese patient with Morquio A syndrome (case report)
por: Hiramatsu, Misako, et al.
Publicado: (2017)

Wiedemann–Steiner Syndrome with a Pathogenic Variant in KMT2A from Taiwan
por: Lee, Chung-Lin, et al.
Publicado: (2021)

The first SHORT syndrome in a Taiwanese boy: A case report and review of the literature
por: Lee, Chung-Lin, et al.
Publicado: (2021)

Otorhinolaryngological Management in Taiwanese Patients with Mucopolysaccharidoses
por: Lee, Chung-Lin, et al.
Publicado: (2021)

Long-Term Cardiovascular Findings in Williams Syndrome: A Single Medical Center Experience in Taiwan
por: Lee, Chung-Lin, et al.
Publicado: (2022)

22q11.2 Deletion Syndrome in Taiwan: Clinical Presentation and Immune System Status of Patients
por: Lee, Chung-Lin, et al.
Publicado: (2023)

Increased Diagnostic Yield of Array Comparative Genomic Hybridization for Autism Spectrum Disorder in One Institution in Taiwan
por: Lee, Chung-Lin, et al.
Publicado: (2021)

Elosulfase alfa in the treatment of mucopolysaccharidosis type IVA: insights from the first managed access agreement
por: Stevens, Bob, et al.
Publicado: (2021)

Functional Independence of Taiwanese Children with Osteogenesis Imperfecta
por: Syu, Yu-Min, et al.
Publicado: (2022)

Clinical characteristics and effects of enzyme replacement therapy with elosulfase alfa in Korean patients with mucopolysaccharidosis type IVA
por: Lee, Seung Hoon, et al.
Publicado: (2022)

Overcoming the barriers to diagnosis of Morquio A syndrome
por: Bhattacharya, Kaustuv, et al.
Publicado: (2014)

Normalization of glycosaminoglycan-derived disaccharides detected by tandem mass spectrometry assay for the diagnosis of mucopolysaccharidosis
por: Lin, Hsiang-Yu, et al.
Publicado: (2019)

Rapid Weight Loss and Severe Failure to Thrive Mimicking Lipodystrophy Syndrome in a 1-Year-Old Taiwanese Girl with Costello Syndrome
por: Syu, Yu-Min, et al.
Publicado: (2022)

An At-Risk Population Screening Program for Mucopolysaccharidoses by Measuring Urinary Glycosaminoglycans in Taiwan
por: Lin, Hsiang-Yu, et al.
Publicado: (2019)

Effect of Mutated ids Overexpression on IDS Enzyme Activity and Developmental Phenotypes in Zebrafish Embryos: A Valuable Index for Assessing Critical Point-Mutations Associated with Mucopolysaccharidosis Type II Occurrence in Humans
por: Lin, Cheng-Yung, et al.
Publicado: (2020)

Identification and Functional Characterization of IDS Gene Mutations Underlying Taiwanese Hunter Syndrome (Mucopolysaccharidosis Type II)
por: Lin, Hsiang-Yu, et al.
Publicado: (2019)

Nationwide Newborn Screening Program for Mucopolysaccharidoses in Taiwan and an Update of the “Gold Standard” Criteria Required to Make a Confirmatory Diagnosis
por: Chuang, Chih-Kuang, et al.
Publicado: (2021)

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
por: Álvarez, J. Víctor, et al.
Publicado: (2019)

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
por: Cleary, Maureen, et al.
Publicado: (2021)

Quantification of Idua Enzymatic Activity Combined with Observation of Phenotypic Change in Zebrafish Embryos Provide a Preliminary Assessment of Mutated idua Correlated with Mucopolysaccharidosis Type I
por: Lin, Cheng-Yung, et al.
Publicado: (2022)

Updated Confirmatory Diagnosis for Mucopolysaccharidoses in Taiwanese Infants and the Application of Gene Variants
por: Chuang, Chih-Kuang, et al.
Publicado: (2022)

Incidence and treatment of adult femoral fractures with osteogenesis imperfecta: An analysis of a center of 72 patients in Taiwan
por: Lee, Chung-Lin, et al.
Publicado: (2021)

Molecular basis of mucopolysaccharidosis IVA (Morquio A syndrome): A review and classification of GALNS gene variants and reporting of 68 novel variants
por: Zanetti, Alessandra, et al.
Publicado: (2021)

The relationships between urinary glycosaminoglycan levels and phenotypes of mucopolysaccharidoses
por: Lin, Hsiang‐Yu, et al.
Publicado: (2018)

Home infusion with Elosulfase alpha (Vimizim(R)) in a UK Paediatric setting
por: Finnigan, Niamh, et al.
Publicado: (2017)

Spinal involvement in mucopolysaccharidosis IVA (Morquio-Brailsford or Morquio A syndrome): presentation, diagnosis and management
por: Solanki, Guirish A., et al.
Publicado: (2013)

Status of newborn screening and follow up investigations for Mucopolysaccharidoses I and II in Taiwan
por: Chuang, Chih-Kuang, et al.
Publicado: (2018)

Bio-Plex immunoassay measuring the quantity of lysosomal N-acetylgalactosamine-6-sulfatase protein in dried blood spots for the screening of mucopolysaccharidosis IVA in newborn: a pilot study
por: Chuang, Chih-Kuang, et al.
Publicado: (2017)

Functional independence of Taiwanese patients with mucopolysaccharidoses
por: Lee, Chung‐Lin, et al.
Publicado: (2019)

Newborn Screening Program for Mucopolysaccharidosis Type II and Long-Term Follow-Up of the Screen-Positive Subjects in Taiwan
por: Lin, Hsiang-Yu, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_m2u7cf89lklpjke2napdg3gte8