Results 261 to 270 of about 1,474,438 (314)

TrialR: critical enablers and the need for reusable Rare Disease Clinical Trial infrastructure in Western Australia. [PDF]

Orphanet J Rare Dis
MacDonald B, Burmaz M, Baker S, Stevens K, Newell A, Baynam G.   +5 more
europepmc   +1 more source

Real‐World Safety and Effectiveness of JAK Inhibitors in Systemic Sclerosis: A Propensity‐Matched Study from the EUSTAR Cohort

Arthritis Care &Research, Accepted Article.
Objective JAK inhibitors (JAKis) have shown promising effects in early‐phase studies of SSc. We aimed to assess the safety and explore effectiveness of JAKis compared to conventional immunosuppressants in SSc. Methods A longitudinal retrospective study of the EUSTAR cohort was performed.
Stefano Di Donato, Marie‐Elise Truchetet, Marco Minerba, Oliver Distler, Juan José Alegre Sancho, Yolanda Braun‐Moscovici, Christina Bergmann, Petros Sfikakis, Jeska K de Vries‐Bouwstra, Murray Baron, Silvia Bellando‐Randone, Lorenzo Dagna, Christopher P Denton, Madelon C Vonk, Vanessa Smith, Ivan Castellvi, Gabriela Riemekasten, Andra Balanescu, Masataka Kuwana, Maria De Santis, Kamal Solanki, Anastas Batalov, Vahan Mukuchyan, Marco Matucci‐Cerinic, Yannick Allanore, Francesco Del Galdo, Michael Hughes, EUSTAR Collaborators   +27 more
wiley   +1 more source

Case Report of Andersen-Tawil Syndrome: Rare Presentation of a Rare Disease. [PDF]

Ann Indian Acad Neurol
Shakya H, Bhatnagar S, Babel P, Kushwaha S, Dhamija RK.   +4 more
europepmc   +1 more source

One‐Step Microfluidic Manufactured Fucose‐Decorated Sweetosomes Choose the Time and the Road for Their Intracellular Journey to Cancer Treatment

Advanced Healthcare Materials, EarlyView.
Sweetosomes, a new class of fucose‐decorated liposomes, are developed via a one‐step microfluidic process without surface chemistry. This study elucidates their main caveolae‐mediated entry and distinct endosomal trafficking. These nanostructures demonstrate superior endosomal escape, organelle acidity modulation, and prolonged plasma persistence ...
Mattia Tiboni, Mariele Montanari, Shiva Khorshid, Michele Verboni, Andrea Duranti, Simone Lucarini, Daniele Lopez, Annalisa Aluigi, Gianluca Morganti, Michele Menotta, Giovanna Panza, Daniel J. Klionsky, Barbara Canonico, Luca Casettari   +13 more
wiley   +1 more source

Respiratory Organ‐on‐a‐Chip for Disease Modeling: From Architecture to Functional Integration

Advanced Healthcare Materials, EarlyView.
Respiratory organ‐on‐a‐chip (ROC) models capture key mechanical and cellular cues of the human respiratory system, enabling quantitative dissection of disease mechanisms. This review links ROC architectures to disease modeling, functional integration, and commercialization, and proposes a decision framework that aligns model complexity with mechanistic
Jinzhuo Hu, Yongjie Tang, Sidi Liu, Tao Xu, Qian Liu   +4 more
wiley   +1 more source

KLINSE: a comprehensive service model for rare disease information and care management support. [PDF]

Orphanet J Rare Dis
Jäger K, Dannenmann-Stern E, Inbasi S, Grasshoff U, Vossler-Wolf C, Graessner H.   +5 more
europepmc   +1 more source

A Versatile Microfluidic Extrusion‐Based Hydrogel Platform for Self‐Organization and Long‐Term Maintenance of Engineered 3D Lymphatic Endothelium

Advanced Healthcare Materials, EarlyView.
Microfluidic coaxial extrusion generates size‐controlled 3D lymphatic tubes from primary human dermal lymphatic endothelial cells in a defined four‐component matrix. These engineered vessels self‐organize into stable lymphatic endothelium, maintain selective macromolecular permeability for 30 days, and enable direct comparison with blood endothelial ...
Elsa Mazari‐Arrighi, Adeline Boyreau, Laura Chaillot, Wilfried Souleyreau, Laetitia Andrique, Pierre‐Olivier Guichet, Thomas Mathivet, Andreas Bikfalvi, Pierre Nassoy   +8 more
wiley   +1 more source

Enhancing rare disease guideline development with real-world data: a method evaluation. [PDF]

BMC Med Res Methodol
Irvine W, van der Kamp LM, Spivack O, Benachi A, Mesas Burgos C, Bakx R, Gaasterland C, Wijnen R.   +7 more
europepmc   +1 more source

Neutrophil‐Mimetic MRI Enables Ultra‐Early Detection of Vascular Inflammation After Stroke

Advanced Healthcare Materials, EarlyView.
In this study, we developed neutrophil‐mimetic MRI probes that detect ultra‐early neuroinflammation following ischemic stroke by targeting E‐selectin. Within seconds of their injection, these microparticles mimic initial leukocyte adhesion to the activated cerebral endothelium.
Marion Isabelle Morvan, Damien Levard, Eloïse Lemarchand, Amal Boublay, Charly Helaine, Jolan Malherbe, Mikaël Naveau, Didier Goux, Igor Khalin, Ankita Talukdar, Cheun Pelleter, Charlène Jacqmarcq, Erwan Baudron, Isabelle Bardou, Sara Martinez de Lizarrondo, Maxime Gauberti, Denis Vivien, Antoine Philippe Fournier   +17 more
wiley   +1 more source