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The PROPHECI trial: a phase II, double-blind, placebo-controlled, randomized clinical trial for the treatment of pseudoxanthoma elasticum with oral pyrophosphate.
Trials
January 2025
Université Côte d'Azur, CNRS, LP2M, Nice, France.
Background: /aims. Pseudoxanthoma Elasticum (PXE, OMIM 264800) is an autosomal, recessive, metabolic disorder characterized by progressive ectopic calcification in the skin, the vasculature and Bruch's membrane. Variants in the ABCC6 gene are associated with low plasma pyrophosphate (PPi) concentration.
View Article and Find Full Text PDFDisorders of Muscle Mass and Tone.
Vet Clin North Am Equine Pract
January 2025
SVM: Department of Medicine and Epidemiology, University of California, Davis, Tupper Hall 2108, One Shields Avenue, Davis, CA 95616, USA. Electronic address:
Muscle disease has various clinical manifestations that range from exertional and non-exertional rhabdomyolysis, fasciculations, weakness, rigidity, stiffness, gait abnormalities, poor performance, and alterations in muscle mass and tone. Neurogenic disorders and non-neurogenic disorders such as primary muscle disease can cause muscle atrophy and changes in muscle tone. Myotonic disorders can have a genetic (eg, inherited channelopathies) or acquired (eg, electrolyte derangements) origin.
View Article and Find Full Text PDFEffects of intrathecal administration of sodium nitroprusside and nicardipine on cerebral pial microcirculation, cortical tissue oxygenation, and electrocortical activity in the early post-resuscitation period in a porcine cardiac arrest model.
PLoS One
January 2025
Department of Emergency Medicine, Chonnam National University Hospital, Gwangju, Republic of Korea.
Recent studies suggested intrathecal vasodilator administration as a therapy to mitigate post-ischemic cerebral hypoperfusion following cardiac arrest. We examined the effects of two commonly used intrathecal vasodilators, sodium nitroprusside (SNP) and nicardipine, on cerebral pial microcirculation, cortical tissue oxygen tension (PctO2), and electrocortical activity in the early post-resuscitation period using a porcine model of cardiac arrest. Thirty pigs were resuscitated after 14 min of untreated cardiac arrest.
View Article and Find Full Text PDFImine-Based Polymeric Mixed Ionic-Electronic Conductors Featuring Degradability and Biocompatibility for Transient Bioinspired Electronics.
Angew Chem Int Ed Engl
January 2025
Sun Yat-Sen University, School of Material Science and Engineering, Nr.135 Xingang Xi Road, 510275, Guangzhou, CHINA.
Degradable features are highly desirable to advance next-generation organic mixed ionic-electronic conductors (OMIECs) for transient bioinspired artificial intelligence devices.It is highly challenging that OMIECs exhibit excellent mixed ionic-electronic behavior and show degradability simultaneously.Specially,in OMIECs,doping is often a tradeoff between structural disorder and charge carrier mobilities.
View Article and Find Full Text PDFAPP antisense oligonucleotides are effective in rescuing mitochondrial phenotypes in human iPSC-derived trisomy 21 astrocytes.
Alzheimers Dement
January 2025
Department of Neuroscience, City University of Hong Kong, Hong Kong, Hong Kong.
Introduction: Antisense oligonucleotides (ASOs) have shown promise in reducing amyloid precursor protein (APP) levels in neurons, but their effects in astrocytes, key contributors to neurodegenerative diseases, remain unclear. This study evaluates the efficacy of APP ASOs in astrocytes derived from an individual with Down syndrome (DS), a population at high risk for Alzheimer's disease (AD).
Methods: Human induced pluripotent stem cells (hiPSCs) from a healthy individual and an individual with DS were differentiated into astrocytes.
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