Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1111/jgs.13569 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
The expanded French compassionate programme for elexacaftor-tezacaftor-ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study.
Lancet Respir Med
November 2024
Université Paris-Cité, Institut Cochin, CNRS, INSERM, Paris, France; Respiratory Medicine and Cystic Fibrosis National Reference Center, Hôpital Cochin, AP-HP, Paris, France; ERN-Lung CF network, Frankfurt, Germany.
Background: Elexacaftor-tezacaftor-ivacaftor has been approved in Europe for people with cystic fibrosis with at least one F508del CFTR variant. Additionally, it is approved by the US Food and Drug Administration (FDA) for people with cystic fibrosis with at least one of 177 rare variants. The aims of this study were to describe the clinical response to elexacaftor-tezacaftor-ivacaftor for people with cystic fibrosis without a F508del CFTR variant in France and to determine CFTR variant responsiveness to elexacaftor-tezacaftor-ivacaftor based on the observed clinical response.
View Article and Find Full Text PDFAdvantages and Challenges of Platform Trials for Disease Modifying Therapies in Parkinson's Disease.
Mov Disord
September 2024
Sorbonne Université, Institut du Cerveau-Paris Brain Institute - ICM, Assistance Publique Hôpitaux de Paris, Inserm, CNRS, Department of Neurology, CIC Neurosciences, Hôpital Pitié-Salpêtrière, French NS-Park/F-CRIN Network, Paris, France.
Traditional drug development in Parkinson's disease (PD) faces significant challenges because of its protracted timeline and high costs. In response, innovative master protocols are emerging and designed to address multiple research questions within a single overarching protocol. These trials may offer advantages such as increased efficiency, agility in adding new treatment arms, and potential cost savings.
View Article and Find Full Text PDFDevelopment and validation of the DBS-PS (Deep Brain Stimulation-Perception Scale): Assessing parkinsonian patients' expectations to prevent post-operative disappointment?
J Neurol Sci
July 2024
Université de Lille, INSERM, CHU Lille, U1172 - LilNCog - Lille Neuroscience & Cognition, LICEND, NS-PARK/FCRIN Network, Lille, France; CHU Lille, Neurology and Movement Disorders Department, Reference Center for Parkinson's Disease, NS-PARK/FCRIN Network, Lille, France.
Article Synopsis
- Recent studies indicate that understanding the preoperative expectations of Parkinson's disease patients undergoing deep brain stimulation (DBS) can enhance treatment outcomes, but there are limited validated tools to measure these expectations.
- The DBS-PS (Deep Brain Stimulation - Perception Scale) was developed as an 11-item self-administered questionnaire, divided into three domains: expectations for PD, social-life and leisure, and intimate life, and validated through exploratory factor analysis and item response theory.
- The study showed that the DBS-PS has good reliability and validity, making it a practical tool for assessing patient expectations in clinical practice, which may help in cognitive restructuring before surgery.
Multicentric investigations of the role in the disease severity of accelerated phospholipid changes in COVID-19 patient airway.
Microbes Infect
July 2024
Sorbonne Université, Inserm U938, Centre de Recherche Saint-Antoine (CRSA), 75012, Paris, France. Electronic address:
Context: The changes in host membrane phospholipids are crucial in airway infection pathogenesis. Phospholipase A2 hydrolyzes host cell membranes, producing lyso-phospholipids and free fatty acids, including arachidonic acid (AA), which contributes significantly to lung inflammation.
Aim: Follow these changes and their evolution from day 1, day 3 to day 7 in airway aspirates of 89 patients with COVID-19-associated acute respiratory distress syndrome and examine whether they correlate with the severity of the disease.
Progression subtypes in Parkinson's disease identified by a data-driven multi cohort analysis.
NPJ Parkinsons Dis
May 2024
Department of Bioinformatics, Fraunhofer Institute for Algorithms and Scientific Computing (SCAI), Sankt Augustin, Germany.
The progression of Parkinson's disease (PD) is heterogeneous across patients, affecting counseling and inflating the number of patients needed to test potential neuroprotective treatments. Moreover, disease subtypes might require different therapies. This work uses a data-driven approach to investigate how observed heterogeneity in PD can be explained by the existence of distinct PD progression subtypes.
View Article and Find Full Text PDFWant 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!