Cargando…

Cerebrospinal fluid neurofilament light chain levels in CLN2 disease patients treated with enzyme replacement therapy normalise after two years on treatment

Classic late infantile neuronal ceroid lipofuscinosis (CLN2 disease) is caused by a deficiency of tripeptidyl-peptidase-1. In 2017, the first CLN2 enzyme replacement therapy (ERT) cerliponase alfa (Brineura) was approved by the FDA and EMA. The CLN2 disease clinical rating scale (CLN2 CRS) was devel...

Descripción completa

Detalles Bibliográficos
Autores principales:	Iwan, Katharina, Patel, Nina, Heslegrave, Amanda, Borisova, Mina, Lee, Laura, Bower, Rebecca, Mole, Sara E., Mills, Philippa B., Zetterberg, Henrik, Mills, Kevin, Gissen, Paul, Heywood, Wendy E.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	F1000 Research Limited 2022
Materias:	Brief Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8777495/ https://www.ncbi.nlm.nih.gov/pubmed/35106137 http://dx.doi.org/10.12688/f1000research.54556.2

Ejemplares similares

Urine proteomics analysis of patients with neuronal ceroid lipofuscinoses
por: Iwan, Katharina, et al.
Publicado: (2020)

Neurofilament Light in CSF and Plasma Is a Marker of Neuronal Damage in HTLV-1–Associated Myelopathy and Correlates With Neuroinflammation
por: Rosadas, Carolina, et al.
Publicado: (2021)

Increased cerebrospinal fluid soluble TREM2 concentration in Alzheimer’s disease
por: Heslegrave, Amanda, et al.
Publicado: (2016)

Cerebrospinal fluid tau, neurogranin, and neurofilament light in Alzheimer's disease
por: Mattsson, Niklas, et al.
Publicado: (2016)

Neurofilament Light Chain Levels Correlate with Clinical Measures in CLN3 Disease
por: Dang Do, An N., et al.
Publicado: (2020)

Neurofilament light is a treatment‐responsive biomarker in CLN2 disease
por: Ru, Yuanbin, et al.
Publicado: (2019)

Correlation between cerebrospinal fluid and plasma neurofilament light protein in treated HIV infection: results from the COBRA study
por: Alagaratnam, Jasmini, et al.
Publicado: (2021)

New Perspectives in Dried Blood Spot Biomarkers for Lysosomal Storage Diseases
por: Spiewak, Justyna, et al.
Publicado: (2023)

Cerebrospinal fluid and plasma neurofilament light relate to abnormal cognition
por: Osborn, Katie E., et al.
Publicado: (2019)

CSF pro-orexin and amyloid-β38 expression in Alzheimer's disease and frontotemporal dementia
por: Heywood, Wendy E., et al.
Publicado: (2018)

Cerebrospinal fluid neurofilament light chain protein levels in subtypes of frontotemporal dementia
por: Landqvist Waldö, Maria, et al.
Publicado: (2013)

Neurofilament light as a blood biomarker for neurodegeneration in Down syndrome
por: Strydom, Andre, et al.
Publicado: (2018)

Blood and cerebrospinal fluid neurofilament light differentially detect neurodegeneration in early Alzheimer’s disease
por: Andersson, Emelie, et al.
Publicado: (2020)

Neurofilament Light in Cerebrospinal Fluid is Associated With Disease Staging in European Lyme Neuroborreliosis
por: Mens, Helene, et al.
Publicado: (2022)

Niemann–Pick type C disease as proof‐of‐concept for intelligent biomarker panel selection in neurometabolic disorders
por: Papandreou, Apostolos, et al.
Publicado: (2022)

Neurofilament light oligomers in neurodegenerative diseases: quantification by homogeneous immunoassay in cerebrospinal fluid
por: Meda, Francisco J., et al.
Publicado: (2023)

Repurposing of tamoxifen ameliorates CLN3 and CLN7 disease phenotype
por: Soldati, Chiara, et al.
Publicado: (2021)

CLN8 disease caused by large genomic deletions
por: Beesley, Clare, et al.
Publicado: (2016)

Serum and cerebrospinal fluid neurofilament light chain in patients with central nervous system infections caused by varicella-zoster virus
por: Tyrberg, Tobias, et al.
Publicado: (2020)

Neurofilament light predicts neurological outcome after subarachnoid haemorrhage
por: Garland, Patrick, et al.
Publicado: (2021)

Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa, Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular Administration
por: Kim, Aryun, et al.
Publicado: (2020)

An ERG and OCT study of neuronal ceroid lipofuscinosis CLN2 Battens retinopathy
por: Thompson, Dorothy A., et al.
Publicado: (2021)

Neurofilament light chain: defining the analyte
por: Leckey, Claire A., et al.
Publicado: (2022)

Guidelines on the diagnosis, clinical assessments, treatment and management for CLN2 disease patients
por: Mole, Sara E., et al.
Publicado: (2021)

Vascular normalisation as the stepping stone into tumour microenvironment transformation
por: Magnussen, Anette L., et al.
Publicado: (2021)

Correction: An ERG and OCT study of neuronal ceroid lipofuscinosis CLN2 Battens retinopathy
por: Thompson, Dorothy A., et al.
Publicado: (2021)

Assessing Neurofilaments as Biomarkers of Neuroprotection in Progressive Multiple Sclerosis: From the MS-STAT Randomized Controlled Trial
por: Williams, Thomas E., et al.
Publicado: (2022)

Serum neurofilament light levels correlate with severity measures and neurodegeneration markers in autosomal dominant Alzheimer’s disease
por: Sánchez-Valle, Raquel, et al.
Publicado: (2018)

Association of plasma neurofilament light chain with disease activity in chronic inflammatory demyelinating polyradiculoneuropathy
por: Kapoor, Mahima, et al.
Publicado: (2022)

The prognostic significance of early blood neurofilament light chain concentration and magnetic resonance imaging variables in relapse‐onset multiple sclerosis
por: Williams, Thomas, et al.
Publicado: (2022)

Cerebrospinal fluid neurofilament light concentration predicts brain atrophy and cognition in Alzheimer's disease
por: Dhiman, Kunal, et al.
Publicado: (2020)

A new enzyme-linked immunosorbent assay for neurofilament light in cerebrospinal fluid: analytical validation and clinical evaluation
por: Gaetani, Lorenzo, et al.
Publicado: (2018)

A central role for TOR signalling in a yeast model for juvenile CLN3 disease
por: Bond, Michael E., et al.
Publicado: (2015)

Longitudinal measurement of serum neurofilament light in presymptomatic familial Alzheimer’s disease
por: Weston, Philip S. J., et al.
Publicado: (2019)

Plasma p-tau181, neurofilament light chain and association with cognition in Parkinson’s disease
por: Batzu, Lucia, et al.
Publicado: (2022)

Neurofilament light chain and tau concentrations are markedly increased in the serum of patients with sporadic Creutzfeldt-Jakob disease, and tau correlates with rate of disease progression
por: Thompson, Andrew Geoffrey Bourne, et al.
Publicado: (2018)

Plasma neurofilament light chain protein as a predictor of days in delirium and deep sedation, mortality and length of stay in critically ill patients
por: Page, Valerie J, et al.
Publicado: (2022)

Neurofilaments in progressive multiple sclerosis: a systematic review
por: Williams, Thomas, et al.
Publicado: (2020)

Neurofilament light in blood – What more is needed for clinical implementation in multiple sclerosis?
por: Zetterberg, Henrik
Publicado: (2020)

Language Delay in Patients with CLN2 Disease: Could It Support Earlier Diagnosis?
por: Nickel, Miriam, et al.
Publicado: (2023)

Cannot write session to /tmp/vufind_sessions/sess_oqatoc62ph15nbs2r1v1bim07f