Cargando…

Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA()

Mucopolysaccharidosis IVA (MPS IVA) is one of the lysosomal storage diseases. It is caused by the deficiency of N-acetylgalactosamine-6-sulfate sulfatase. Deficiency of this enzyme leads to accumulation of the specific glycosaminoglycans keratan sulfate and chondroitin-6-sulfate. This accumulation h...

Descripción completa

Detalles Bibliográficos
Autores principales:	Chinen, Yasutsugu, Higa, Takeshi, Tomatsu, Shunji, Suzuki, Yasuyuki, Orii, Tadao, Hyakuna, Nobuyuki
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2014
Materias:	Research Paper
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4292891/ https://www.ncbi.nlm.nih.gov/pubmed/25593792 http://dx.doi.org/10.1016/j.ymgmr.2013.11.002

Ejemplares similares

Long-Term Follow-up Posthematopoietic Stem Cell Transplantation in a Japanese Patient with Type-VII Mucopolysaccharidosis
por: Orii, Kenji, et al.
Publicado: (2020)

Assessment of Activity of Daily Life in Mucopolysaccharidosis Type II Patients with Hematopoietic Stem Cell Transplantation
por: Suzuki, Yasuyuki, et al.
Publicado: (2020)

Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management
por: Sawamoto, Kazuki, et al.
Publicado: (2020)

Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacy
por: Chen, Hui, et al.
Publicado: (2021)

Bone Growth Induction in Mucopolysaccharidosis IVA Mouse
por: Rintz, Estera, et al.
Publicado: (2023)

Mucopolysaccharidosis IVA: Current Disease Models and Drawbacks
por: Leal, Andrés Felipe, et al.
Publicado: (2023)

Pathophysiology of Hip Disorders in Patients with Mucopolysaccharidosis IVA
por: Wang, Zhigang, et al.
Publicado: (2020)

Development of Substrate Degradation Enzyme Therapy for Mucopolysaccharidosis IVA Murine Model
por: Sawamoto, Kazuki, et al.
Publicado: (2019)

Cochlear implantation in a patient with mucopolysaccharidosis IVA
por: Nagao, Kyoko, et al.
Publicado: (2019)

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

Characterization of New Proteomic Biomarker Candidates in Mucopolysaccharidosis Type IVA
por: Álvarez, Víctor J., et al.
Publicado: (2020)

Diagnosing mucopolysaccharidosis IVA
por: Wood, Timothy C., et al.
Publicado: (2013)

Safety Study of Sodium Pentosan Polysulfate for Adult Patients with Mucopolysaccharidosis Type II
por: Orii, Kenji, et al.
Publicado: (2019)

Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model
por: Sawamoto, Kazuki, et al.
Publicado: (2020)

Bone mineral density in mucopolysaccharidosis IVB
por: Kubaski, Francyne, et al.
Publicado: (2016)

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

Atypical presentation of mucopolysaccharidosis type IVA
por: Rush, Eric T.
Publicado: (2016)

Growth charts for patients with Hunter syndrome()
por: Patel, Pravin, et al.
Publicado: (2014)

Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy
por: Piechnik, Matthew, et al.
Publicado: (2022)

Comparison of Liquid Chromatography–Tandem Mass Spectrometry and Sandwich ELISA for Determination of Keratan Sulfate in Plasma and Urine
por: Hintze, Jonathan P., et al.
Publicado: (2011)

Natural history and clinical assessment of Taiwanese patients with mucopolysaccharidosis IVA
por: Lin, Hsiang-Yu, et al.
Publicado: (2014)

Clinical, radiologic, and genetic features of Korean patients with Mucopolysaccharidosis IVA
por: Lee, Na Hee, et al.
Publicado: (2012)

Determination of genotypic and clinical characteristics of Colombian patients with mucopolysaccharidosis IVA
por: Tapiero-Rodriguez, Sandra M, et al.
Publicado: (2018)

Mucopolysaccharidosis Type IVA: Extracellular Matrix Biomarkers in Cardiovascular Disease
por: Montavon, Brittany, et al.
Publicado: (2022)

Endoscopic and Image Analysis of the Airway in Patients with Mucopolysaccharidosis Type IVA
por: Lee, Yi-Hao, et al.
Publicado: (2023)

Newborn screening for mucopolysaccharidoses: Measurement of glycosaminoglycans by LC-MS/MS
por: Stapleton, Molly, et al.
Publicado: (2020)

Development of Bone Targeting Drugs
por: Stapleton, Molly, et al.
Publicado: (2017)

Erratum: Clinical, radiologic, and genetic features of Korean patients with Mucopolysaccharidosis IVA
por: Lee, Na Hee, et al.
Publicado: (2013)

Muskoskeletal manifestations of mild form of mucopolysaccharidosis iva - a clinical case
por: Ganeva, Margarita, et al.
Publicado: (2014)

Shutdown of ER-associated degradation pathway rescues functions of mutant iduronate 2-sulfatase linked to mucopolysaccharidosis type II
por: Osaki, Yosuke, et al.
Publicado: (2018)

Impact of enzyme replacement therapy and hematopoietic stem cell therapy on growth in patients with Hunter syndrome
por: Patel, Pravin, et al.
Publicado: (2014)

Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA
por: Hendriksz, Christian J., et al.
Publicado: (2012)

Heart and Cardiovascular Involvement in Patients with Mucopolysaccharidosis Type IVA (Morquio-A Syndrome)
por: Kampmann, Christoph, et al.
Publicado: (2016)

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

Chondroitin sulfate disaccharide is a specific and sensitive biomarker for mucopolysaccharidosis type IVA
por: Chin, Sharon J., et al.
Publicado: (2020)

Respiratory Dysfunction in Children and Adolescents with Mucopolysaccharidosis Types I, II, IVA, and VI
por: Tulebayeva, Assel, et al.
Publicado: (2020)

Enhanced Efficiency of the Basal and Induced Apoptosis Process in Mucopolysaccharidosis IVA and IVB Human Fibroblasts
por: Brokowska, Joanna, et al.
Publicado: (2023)

Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome
por: Tomatsu, Shunji, et al.
Publicado: (2015)

LRBA is Essential for Allogeneic Responses in Bone Marrow Transplantation
por: Park, Mi Young, et al.
Publicado: (2016)

Development of a newborn screening tool for mucopolysaccharidosis type I based on bivariate normal limits: Using glycosaminoglycan and alpha‐L‐iduronidase determinations on dried blood spots to predict symptoms
por: Langan, Thomas J., et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_f11bpoqhpd164l9eev0a8vlipa