Results 91 to 100 of about 4,617 (234)

A Review of Tafamidis for the Treatment of Transthyretin-Related Amyloidosis [PDF]

open access: yes, 2015
Transthyretin (TTR)-related amyloidosis (ATTR) is a devastating disease which affects a combination of organs including the heart and the peripheral nerves, and which has a fatal outcome if not treated within a average of 10 years.
Benson, Merril D.   +1 more
core   +1 more source

Diabetic Peripheral Neuropathy: Molecular Staging, Risk Factors, Therapeutics, and Emerging Trends

open access: yesMed Research, EarlyView.
The heterogeneous landscape of DPN can be unified through a tripartite pathogenic model encompassing progressive stages of metabolic dysregulation, chronic inflammation, and overt neuronal damage. Within this framework, six clinical subtypes were identified, namely, hyperglycemia‐driven, dyslipidemia‐driven, inflammation‐driven, dysvascularity‐driven ...
Xiaofeng Dai, Mingze Tang
wiley   +1 more source

Insensibilité congénitale à la douleur

open access: yesThe Pan African Medical Journal, 2014
L'insensibilité congénitale à la douleur (ICD), est une affection rare de transmission autosomique récessive, caractérisée par une absence congénitale de la perception de la douleur.
Kawtar Inani, Fatimazahra Mernissi
doaj   +1 more source

Peripheral Blood Cell Gene Expression Diagnostic for Identifying Symptomatic Transthyretin Amyloidosis Patients: Male and Female Specific Signatures [PDF]

open access: yes, 2016
BACKGROUND: Early diagnosis of familial transthyretin (TTR) amyloid diseases remains challenging because of variable disease penetrance. Currently, patients must have an amyloid positive tissue biopsy to be eligible for disease-modifying therapies ...
Buxbaum, J.   +7 more
core   +1 more source

Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male and Female Patients

open access: yesMuscle &Nerve, EarlyView.
This exploratory analysis of data from the NEURO‐TTRansform trial evaluated sex‐specific responses in patients with ATTRv‐PN who had received eplontersen in NEURO‐TTRansform versus a historical placebo. Eplontersen can lead to substantial decreases in percentage serum TTR levels, arrest neuropathy impairment, and improve patient quality of life ...
Márcia Waddington Cruz   +11 more
wiley   +1 more source

Uncoupling neurotrophic function from nociception of nerve growth factor: what can be learned from a rare human disease?

open access: yesNeural Regeneration Research, 2019
Nerve growth factor (NGF) is a powerful trophic factor that provides essential support for the survival and differentiation of sympathetic and sensory neurons during development.
Kijung Sung, Wanlin Yang, Chengbiao Wu
doaj   +1 more source

Effect of Capsaicin (8%) Topical System on Sensory Function in Patients With Diabetic Peripheral Neuropathy: Analysis of the PACE Study

open access: yesMuscle &Nerve, EarlyView.
This graphical abstract provides a summary of the analysis of the PACE study that evaluated the effect of high‐concentration capsaicin topical system (HCCTS) on sensory function in patients with DPN. From left to right: Progression of untreated DPN; Research question for current analysis; Outcomes used to evaluate sensory function; Intervention; Main ...
Nathaniel P. Katz   +4 more
wiley   +1 more source

Compound heterozygous mutations in glycyl-tRNA synthetase are a proposed cause of systemic mitochondrial disease [PDF]

open access: yes, 2014
Background Glycyl-tRNA synthetase (GARS) is an aminoacyl-tRNA synthetase (ARS) that links the amino acid glycine to its corresponding tRNA prior to protein translation and is one of three bifunctional ARS that are active within ...
Amanda Smith   +9 more
core   +1 more source

What Role Does the Central Nervous System Play in Refractory LUTS, and What Are the Therapeutic Implications? ICI‐RS 2025

open access: yesNeurourology and Urodynamics, EarlyView.
ABSTRACT Aims While many patients with lower urinary tract symptoms (LUTS) improve by treating peripheral causes, a substantial proportion continue to experience symptoms despite apparently successful interventions. Central nervous system (CNS) mechanisms could potentially contribute to persisting symptoms after the initial peripheral cause has been ...
Mathijs M. de Rijk   +7 more
wiley   +1 more source

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