Cargando…

Impact of the Timing of Enzyme Replacement Therapy Initiation and Cognitive Impairment Status on Outcomes for Patients with Mucopolysaccharidosis II (MPS II) in the United States: A Retrospective Chart Review

Background: Mucopolysaccharidosis II (MPS II; Hunter syndrome; OMIM 309900) is a rare, X-linked, lysosomal storage disease caused by deficient iduronate-2-sulfatase activity. Accumulation of glycosaminoglycans results in multisystemic disease manifestations, which may include central nervous system...

Descripción completa

Detalles Bibliográficos
Autores principales:	Yee, Karen S., Alexanderian, David, Feng, Yidie, Ren, Xiaowei, Schweikert, Bernd, Ayodele, Olulade
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Columbia Data Analytics, LLC 2022
Materias:	Neurological Diseases
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9424538/ https://www.ncbi.nlm.nih.gov/pubmed/36168594 http://dx.doi.org/10.36469/001c.36540

Ejemplares similares

Clinical Characteristics and Healthcare Resource Utilization for Patients with Mucopolysaccharidosis II (MPS II) in the United States: A Retrospective Chart Review
por: Ayodele, Olulade, et al.
Publicado: (2022)

Mucopolysaccharidosis: Cardiologic features and effects of enzyme-replacement therapy in 24 children with MPS I, II and VI
por: Brands, Marion M. M. G., et al.
Publicado: (2012)

The Value of Case Reports in Systematic Reviews from Rare Diseases. The Example of Enzyme Replacement Therapy (ERT) in Patients with Mucopolysaccharidosis Type II (MPS-II)
por: Sampayo-Cordero, Miguel, et al.
Publicado: (2020)

Effects of enzyme replacement therapy on growth in patients with mucopolysaccharidosis type II
por: Schulze-Frenking, G., et al.
Publicado: (2010)

Enzyme replacement therapy for Mucopolysaccharidosis Type I among patients followed within the MPS Brazil Network
por: Dornelles, Alícia Dorneles, et al.
Publicado: (2014)

Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II
por: Seo, Joo-Hyun, et al.
Publicado: (2021)

Immunological evaluation of patients with mucopolysaccharidosis (MPS)
por: Aranda, Carolina, et al.
Publicado: (2015)

Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter Syndrome)
por: Sohn, Young Bae, et al.
Publicado: (2013)

Patient's weight can decide about spending millions on enzyme replacement therapy in MPS II
por: Bik-Multanowski, Miroslaw, et al.
Publicado: (2015)

Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)
por: Cho, Sung Yoon, et al.
Publicado: (2014)

Recovery of Vision following Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type II, Hunter Syndrome
por: Yamanishi, Ryutaro, et al.
Publicado: (2019)

Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis of Preclinical and Clinical Data
por: Giugliani, Roberto, et al.
Publicado: (2021)

Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study
por: Muenzer, Joseph, et al.
Publicado: (2023)

Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
por: Wraith, J. Edmond, et al.
Publicado: (2007)

Natural progression of cardiac features and long-term effects of enzyme replacement therapy in Taiwanese patients with mucopolysaccharidosis II
por: Lin, Hsiang-Yu, et al.
Publicado: (2021)

Osteoarthropathy in mucopolysaccharidosis type II
por: NASCU, IOANA, et al.
Publicado: (2013)

Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients
por: Giugliani, R., et al.
Publicado: (2017)

Glycosaminoglycan signatures in body fluids of mucopolysaccharidosis type II mouse model under long-term enzyme replacement therapy
por: Maccari, Francesca, et al.
Publicado: (2022)

Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?
por: Horgan, Claire, et al.
Publicado: (2022)

Differences in MPS I and MPS II Disease Manifestations
por: Hampe, Christiane S., et al.
Publicado: (2021)

Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II
por: Cho, Sung Yoon, et al.
Publicado: (2015)

Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)
por: Muenzer, Joseph, et al.
Publicado: (2021)

Cervical spondylolisthesis in mucopolysaccharidosis type II
por: Rossi, Alessandro, et al.
Publicado: (2022)

Genotype‐phenotype relationships in mucopolysaccharidosis type I (MPS I): Insights from the International MPS I Registry
por: Clarke, Lorne A., et al.
Publicado: (2019)

Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series
por: Lampe, Christina, et al.
Publicado: (2014)

Perioperative airway management for aortic valve replacement in an adult with mucopolysaccharidosis type II (Hunter syndrome)
por: Suzuki, Kazuchika, et al.
Publicado: (2018)

Review of the use of idursulfase in the treatment of mucopolysaccharidosis II
por: Burrow, T Andrew, et al.
Publicado: (2008)

Biomarkers in patients with mucopolysaccharidosis type II and IV
por: Fujitsuka, Honoka, et al.
Publicado: (2019)

Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry
por: D’Aco, Kristin, et al.
Publicado: (2012)

Parents’ experiences of living with, and caring for children, adolescents and young adults with Mucopolysaccharidosis (MPS)
por: Somanadhan, S., et al.
Publicado: (2016)

Sexual behaviour in a murine model of mucopolysaccharidosis type I (MPS I)
por: Barbosa Mendes, Ana, et al.
Publicado: (2019)

Mucopolysaccharidosis (MPS): Review of the literature and case series of five pediatric dental patients
por: Hirst, Lorna, et al.
Publicado: (2021)

Safety outcomes and patients’ preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond
por: Toscano, Antonio, et al.
Publicado: (2023)

Growth charts for patients with Sanfilippo syndrome (Mucopolysaccharidosis type III)
por: Muschol, Nicole M., et al.
Publicado: (2019)

Cardiac features and effects of enzyme replacement therapy in Taiwanese patients with Mucopolysaccharidosis IVA
por: Lin, Hsiang-Yu, et al.
Publicado: (2018)

Intrathecal enzyme replacement therapy reverses cognitive decline in mucopolysaccharidosis type I
por: Nestrasil, Igor, et al.
Publicado: (2017)

Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I
por: Ghosh, Arunabha, et al.
Publicado: (2019)

Novel approach to idursulfase and laronidase desensitization in type 2 and type 1 S mucopolysaccharidosis (MPS)
por: Spataro, Federico, et al.
Publicado: (2022)

Neurological, neurobehavioral, and radiological alterations in patients with mucopolysaccharidosis III (Sanfilippo's syndrome) in Brazil
por: do Valle, Daniel Almeida, et al.
Publicado: (2022)

Clinical Presentation of Mucopolysaccharidosis Type II (Hunter's Syndrome)
por: Chinawa, JM, et al.
Publicado: (2012)

Cannot write session to /tmp/vufind_sessions/sess_b69lgj8fnesisisekn83jkhck7