Results 111 to 120 of about 1,024,087 (311)

Unraveling 4‐Phenylbutyrate's Therapeutic Role in SLC6A1 Disorders: Pharmacochaperoning Over HDAC Inhibition

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Variants in SLC6A1, encoding the GABA transporter 1 (GAT‐1), cause epilepsy, autism spectrum disorder, and developmental delay via loss of GABA uptake, impaired trafficking, and ER retention. We previously found that 4‐Phenylbutyrate (PBA), an FDA‐approved drug, restores GABA uptake and reduces seizures in SLC6A1‐related disorders ...
Melissa B. DeLeeuw   +5 more
wiley   +1 more source

Ventilation inhomogeneity in patients with cystic fibrosis measured by electrical impedance tomography [PDF]

open access: yes, 2012
Objective: The distribution of ventilation within the lung is inhomogeneous. We hypothesized that the degree of inhomogeneity in patients with cystic fibrosis (CF) differs from that in healthy subjects.
Zhao, Zhanqi   +3 more
core   +1 more source

Televisit with TytoHome™ device in medically complex child in long-term mechanical ventilation: a pilot study

open access: yesItalian Journal of Pediatrics
Background During the pandemic, the pneumology team at Bambino Gesù Children’s Hospital highlighted that telemedicine was a valuable tool for remotely managing the medical needs of children with medical complexity (CMC).
Alessandro Onofri   +5 more
doaj   +1 more source

Physical activity and activity space in patients with pulmonary fibrosis not prescribed supplemental oxygen

open access: yesBMC Pulmonary Medicine, 2017
Background Patients with pulmonary fibrosis (PF) have impaired quality of life, and research suggests that dyspnea and physical activity are primary drivers. As PF progresses, some patients notice the disease “shrinks their worlds”. The objective of this
Elisabeth Dowling Root   +10 more
doaj   +1 more source

Plasma EV Proteomics Identifies ECM Remodeling and Inflammatory Proteins LUM and C7 as Candidate Biomarkers in FSHD

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Facioscapulohumeral muscular dystrophy (FSHD) is one of the most debilitating and common muscular dystrophies. Despite its severity, no approved therapy exists for FSHD patients. However, several therapeutic candidates are currently under development, and some have recently entered clinical trials, marking the need for reliable ...
Mustafa Bilal Bayazit   +11 more
wiley   +1 more source

Lung function, symptoms and inflammation during exacerbations of non-cystic fibrosis bronchiectasis: a prospective observational cohort study. [PDF]

open access: yes, 2015
Exacerbations of non-cystic fibrosis bronchiectasis cause significant morbidity but there are few detailed data on their clinical course and associated physiological changes.
Hurst, JR   +5 more
core  

Glucagon‐Like Peptide‐1 Receptor Agonists in Multiple Sclerosis: A Topical Narrative Review

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT The glucagon‐like peptide‐1 (GLP‐1) receptor agonists (RAs) are a relatively new class of drugs that were initially approved by the US Food and Drug Administration to improve glycemic control in patients with type 2 diabetes mellitus and, subsequently, weight reduction in patients with obesity.
Audrey Y. Chang, Farrah J. Mateen
wiley   +1 more source

Elevated Paracellular Glucose Flux across Cystic Fibrosis Airway Epithelial Monolayers Is an Important Factor for Pseudomonas aeruginosa Growth. [PDF]

open access: yes, 2013
People with cystic fibrosis (CF) who develop related diabetes (CFRD) have accelerated pulmonary decline, increased infection with antibiotic-resistant Pseudomonas aeruginosa and increased pulmonary exacerbations.
James P Garnett   +27 more
core   +1 more source

Artificial Intelligence in Systemic Sclerosis: Clinical Applications, Challenges, and Future Directions

open access: yesArthritis Care &Research, EarlyView.
Systemic sclerosis (SSc) is a rare autoimmune disease defined by immune dysregulation, vasculopathy, and progressive fibrosis of the skin and internal organs. Despite advances in care, major complications such as interstitial lung disease (ILD) and myocardial involvement remain the leading causes of morbidity and mortality.
Cristiana Sieiro Santos   +2 more
wiley   +1 more source

Fighting time: A qualitative analysis on the impact of lung transplantation in adults with Cystic Fibrosis [PDF]

open access: yes, 2001
During the last decade lung transplantation has become an option for those with end-stage respiratory disease, including Cystic Fibrosis. However, there appears to have been very little written about the experiences of those who are on the lung ...
McCarthy, Janet Mary
core   +2 more sources

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