Correction: Qualitative analysis of patient interviews on the burden of neuronopathic Gaucher disease in Japan

Correction: Qualitative analysis of patient interviews on the burden of neuronopathic Gaucher disease in Japan

Bibliographic Details
Main Authors:	Yuta Koto, Aya Narita, Shinichi Noto, Midori Ono, Anna Lissa Hamada, Norio Sakai
Format:	Article
Language:	English
Published:	BMC 2023-03-01
Series:	Orphanet Journal of Rare Diseases
Online Access:	https://doi.org/10.1186/s13023-023-02659-9

Similar Items

Qualitative analysis of patient interviews on the burden of neuronopathic Gaucher disease in Japan
by: Yuta Koto, et al.
Published: (2022-07-01)

Burden of caregivers of patients with neuronopathic and non-neuronopathic Gaucher disease in Japan: A survey-based study
by: Yuta Koto, et al.
Published: (2023-09-01)

Development and evaluation of a patient-reported outcome measure specific for Gaucher disease with or without neurological symptoms in Japan
by: Aya Narita, et al.
Published: (2024-01-01)

Neuronopathic Gaucher disease: Beyond lysosomal dysfunction
by: Nohela B. Arévalo, et al.
Published: (2022-08-01)

Identification of a biomarker in cerebrospinal fluid for neuronopathic forms of Gaucher disease.
by: Hila Zigdon, et al.
Published: (2015-01-01)

Successful therapy for protein-losing enteropathy caused by chronic neuronopathic Gaucher disease
by: A.A. Mhanni, et al.
Published: (2016-03-01)

A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative
by: Tanya Collin-Histed, et al.
Published: (2023-07-01)

Neuronopathic Gaucher disease models reveal defects in cell growth promoted by Hippo pathway activation
by: Daria Messelodi, et al.
Published: (2023-04-01)

Thirty-year clinical outcomes after haematopoietic stem cell transplantation in neuronopathic Gaucher disease
by: Aimee Donald, et al.
Published: (2022-06-01)

Neuroinflammation in neuronopathic Gaucher disease: Role of microglia and NK cells, biomarkers, and response to substrate reduction therapy
by: Chandra Sekhar Boddupalli, et al.
Published: (2022-08-01)

Viable neuronopathic Gaucher disease model in Medaka (Oryzias latipes) displays axonal accumulation of alpha-synuclein.
by: Norihito Uemura, et al.
Published: (2015-04-01)

Impact on physical, social, and family functioning of patients with metachromatic leukodystrophy and their family members in Japan: A qualitative study
by: Yuta Koto, et al.
Published: (2024-03-01)

A multifaceted evaluation of microgliosis and differential cellular dysregulation of mammalian target of rapamycin signaling in neuronopathic Gaucher disease
by: Zhenting Zhang, et al.
Published: (2022-09-01)

Rapid and long‐lasting efficacy of high‐dose ambroxol therapy for neuronopathic Gaucher disease: A case report and literature review
by: Kanako Higashi, et al.
Published: (2024-04-01)

Systemic enzyme delivery by blood-brain barrier-penetrating SapC-DOPS nanovesicles for treatment of neuronopathic Gaucher disease
by: Ying Sun, et al.
Published: (2020-05-01)

An increase in ER stress and unfolded protein response in iPSCs-derived neuronal cells from neuronopathic Gaucher disease patients
by: Tanapat Pornsukjantra, et al.
Published: (2024-04-01)

Elevated Dkk1 Mediates Downregulation of the Canonical Wnt Pathway and Lysosomal Loss in an iPSC Model of Neuronopathic Gaucher Disease
by: Manasa P. Srikanth, et al.
Published: (2020-12-01)

Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes
by: Massaro, G, et al.
Published: (2020)

Experiences of mothers of long-term surviving patients with cerebral adrenoleukodystrophy: a qualitative study
by: Yuta Koto, et al.
Published: (2024-10-01)

Neuronopathic Types of Mucopolysaccharidoses: Pathogenesis and Emerging Treatments
by: L. A. Osipova, et al.
Published: (2015-11-01)

Investigating the neurobehavioral symptoms of neuronopathic Hunter syndrome
by: Nathan Grant
Published: (2020-03-01)

Response to: Investigating the neurobehavioral symptoms of neuronopathic Hunter syndrome
by: J.B. Eisengart, et al.
Published: (2020-03-01)

Development and validation of a disease-specific quality of life scale for adult patients with Fabry disease in Japan
by: Yuta Koto, et al.
Published: (2022-11-01)

Elevated LysoGb3 Concentration in the Neuronopathic Forms of Mucopolysaccharidoses
by: Galina Baydakova, et al.
Published: (2020-03-01)

Gaucher Disease and Gaucher Cells
by: Sevgi Gözdaşoğlu
Published: (2015-05-01)

Analysis of caregiver perspectives on patients with mucopolysaccharidosis II treated with pabinafusp alfa: results of qualitative interviews in Japan
by: Kimitoshi Nakamura, et al.
Published: (2024-03-01)

Gaucher Cells or Pseudo-Gaucher Cells: That's the Question
by: Deniz Gören Şahin, et al.
Published: (2014-12-01)

Editorial: Neuronopathic lysosomal storage diseases - specific neuronal characteristics and therapeutic approaches
by: Stefanie Flunkert, et al.
Published: (2022-11-01)

Supporters' experiences of sensory characteristics of children with profound intellectual and multiple disabilities in after‐school daycare centres: A qualitative study
by: Yuta Koto, et al.
Published: (2023-12-01)

Gaucher disease.
by: Butters, T
Published: (2007)

GAUCHER’S DISEASE
by: O. S. Gundobina, et al.
Published: (2013-11-01)

Effect of Ambroxol chaperone therapy on Glucosylsphingosine (Lyso-Gb1) levels in two Canadian patients with type 3 Gaucher disease
by: Behshad Charkhand, et al.
Published: (2019-09-01)

An Overview of Gaucher Disease
by: Daniela Anahí Méndez-Cobián, et al.
Published: (2024-12-01)

Women with Gaucher Disease
by: Maria del Mar Meijon-Ortigueira, et al.
Published: (2024-03-01)

Gaucher Disease for Hematologists
by: Gül Nihal Özdemir, et al.
Published: (2022-05-01)

Doença de Gaucher.
by: L P Trindade e Silva, et al.
Published: (2007-04-01)

Enfermedad de gaucher
by: Magaly Mendoza-Quispe
Published: (2018-04-01)

PB2551: GAUCHER DISEASE CURRICULUM: INCORPORATING GAUCHER DISEASE IN THE FIELD OF HEMATOLOGY
by: Argiris Symeonidis, et al.
Published: (2023-08-01)

Advancing clinical development for neuronopathic Hunter syndrome through a quantitatively‐driven reverse translation framework
by: Robert D. Latzman, et al.
Published: (2024-04-01)

Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment
by: Karen S. Yee, et al.
Published: (2024-04-01)