Death in the late stage of Duchenne muscular dystrophy is most frequently a consequence of respiratory failure. Since muscles of ventilation become weakened the bellows mechanism fails insidiously. Patients exhibit symptoms of ventilatory insufficiency, the first to be noticed are those associated with CO2 retention: dyspnea, nightmares, increased heart rate, and increased blood pressure. Ten patients with late stage Duchenne muscular dystrophy have been supplied with mechanical aid for ventilatory assistance. The age of onset of respiratory distress needing mechanical assistance varied from 10 to 20 years. Meaningful survival after allegedly reaching the end stage has been from 2 to 7.5 years with an average of 3.4 years. With a caring family, these patients can have a meaningful life, even though they require continuous mechanical ventilatory aid.

Download full-text PDF

Source

Publication Analysis

Top Keywords

late stage
12
stage duchenne
12
duchenne muscular
12
muscular dystrophy
12
patients late
8
mechanical
4
mechanical ventilation
4
patients
4
ventilation patients
4
stage
4

Similar Publications

Intermolecular oxidative N-N bond formation reactions are quite challenging and are largely uncharted. N-N linked dimeric indolosesquiterpene alkaloids represent an underexplored class of natural products, and strategies for direct dehydrogenative N-N bond formation are limited. Here, we have reported that a late-stage visible-light photoredox catalysis facilitates N-N bond formation, leading to the total syntheses of atropo-diastereomers dixiamycins A () and B ().

View Article and Find Full Text PDF

Utility of physiologically based pharmacokinetic modeling in predicting and characterizing clinical drug interactions.

Drug Metab Dispos

January 2025

Pharmacokinetics, Dynamics, Metabolism and Bioanalytics, Merck & Co, Inc, Boston, Massachusetts. Electronic address:

Physiologically based pharmacokinetic (PBPK) modeling is a mechanistic dynamic modeling approach that can be used to predict or retrospectively describe changes in drug exposure due to drug-drug interactions (DDIs). With advancements in commercially available PBPK software, PBPK DDI modeling has become a mainstream approach from early drug discovery through to late-stage drug development and is often used to support regulatory packages for new drug applications. This Minireview will briefly describe the approaches to predicting DDI using PBPK and static modeling approaches, the basic model structures and features inherent to PBPK DDI models, and key examples where PBPK DDI models have been used to describe complex DDI mechanisms.

View Article and Find Full Text PDF

Characterization of Subcutaneous and Visceral De-differentiated Fat Cells.

Mol Metab

January 2025

Peking-Tsinghua Center for Life Sciences, Beijing, China 100871; PKU-IDG/McGovern Institute for Brain Research, Beijing, China 100871; School of Psychological and Cognitive Sciences, Peking University, Beijing, China 100871; Beijing Key Laboratory of Behavior and Mental Health, Beijing, China 100871; Biosciences and Nutrition Unit, Department of Medicine Huddinge, Karolinska Institute, Huddinge, Sweden 14183. Electronic address:

The capacity of mature adipocytes to de-differentiate into fibroblast-like cells has been demonstrated in vitro and a few, rather specific in vivo conditions. A detailed comparison between de-differentiated fat (DFAT) cells and adipose stem and progenitor cells (ASPCs) from different adipose depots is yet to be conducted. Moreover, whether de-differentiation of mature adipocytes from classical subcutaneous and visceral depots occurs under physiological conditions remains unknown.

View Article and Find Full Text PDF

Unveiling the Content of Frontal Feedback in Challenging Object Recognition.

Neuroimage

January 2025

School of Electrical and Computer Engineering, College of Engineering, University of Tehran, Tehran, Iran; School of Cognitive Sciences, Institute for Research in Fundamental Sciences, Tehran, Iran. Electronic address:

Object recognition under challenging real-world conditions, including partial occlusion, remains an enduring focus of investigation in cognitive visual neuroscience. This study addresses the insufficiently elucidated neural mechanisms and temporal dynamics involved in this complex process, concentrating on the persistent challenge of recognizing objects obscured by occlusion. Through the analysis of human EEG data, we decode feedback characteristics within frontotemporal networks, uncovering intricate neural mechanisms during occlusion coding, with a specific emphasis on processing complex stimuli such as occluded faces.

View Article and Find Full Text PDF

Damage sensing through TLR9 regulates inflammatory and antiviral responses during influenza infection.

Mucosal Immunol

January 2025

Division of Pulmonary, Allergy, Critical Care and Sleep Medicine, University of Pittsburgh Medical Center Pittsburgh PA USA; Section of Pulmonary, Critical Care and Sleep Medicine, Yale School of Medicine New Haven CT USA. Electronic address:

Host response aimed at eliminating the infecting pathogen, as well as the pathogen itself, can cause tissue injury. Tissue injury leads to the release of a myriad of cellular components including mitochondrial DNA, which the host senses through pattern recognition receptors. How the sensing of tissue injury by the host shapes the anti-pathogen response remains poorly understood.

View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!