Results 171 to 180 of about 58,664 (301)

Can long-acting growth hormone improve adherence in pediatric growth hormone deficiency? [PDF]

Endocrine
Rodaro C, Tamaro G, Fachin A, Nicolardi F, Tommasi S, Tornese G.   +5 more
europepmc   +1 more source

Re‐Purposing Sapropterin (Kuvan) for ACTA2‐Related Multisystemic Smooth Muscle Dysfunction Syndrome: A Translational Mechanistic and First‐In‐Human Therapeutic Report

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Multisystemic smooth muscle dysfunction syndrome (MSMDS) is an ultra‐rare, ACTA2‐related disorder characterized by severe cerebrovascular disease, aortic aneurysms, and smooth muscle dysfunction. Using molecular dynamics simulations and in silico drug screening, we identified that sapropterin dihydrochloride (Kuvan) is a candidate capable of ...
Moran Hausman‐Kedem, Noa Bielopolski, Vijai Krishnan, Shane Wald‐Altman, Shelly I Shrian, Omer Bar‐Yosef, Livia Kapusta, Claire Shamber, Patricia L. Musolino, Miguel Weil   +9 more
wiley   +1 more source

Response to: The Long Game in Ulcerative Colitis: Mirikizumab's Four-Year Outcomes. [PDF]

Inflamm Bowel Dis
Clemow DB.
europepmc   +1 more source

Augmenting and Assaying Nav1.1 Protein Quantity for Dravet Syndrome Therapy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Dravet Syndrome (DS) is a developmental and epileptic encephalopathy predominantly caused by heterozygous loss‐of‐function variants in SCN1A, which encodes Nav1.1. Conserved upstream open reading frames (uORFs) in SCN1A were validated to regulate translation in reporter assays, demonstrating the therapeutic viability of increasing Nav1.1 from ...
Aiswarya Saravanan, Jordan Safran, Anna Loughman, Grace Lin, Shivanshi Vaid, Dana Allababidi, Peter M. Tessier, Louis T. Dang   +7 more
wiley   +1 more source

Clinical evidence and implementation guidance for biosimilar switching in ankylosing spondylitis. [PDF]

Front Pharmacol
Cai L, Chen X, Fang Y, Li L, Liu T, Gan Z, Zuo Z.   +6 more
europepmc   +1 more source

Associations of Lifestyle‐Related Factors and Psoriatic Arthritis Disease Activity: The Dutch South West Psoriatic Arthritis Study

Arthritis Care &Research, EarlyView.
Objective This study aims to investigate lifestyle‐related factors in patients with psoriatic arthritis (PsA) and their association with disease activity measurements. Methods This multicenter cohort included 938 patients who were newly diagnosed with PsA between 2013 and 2023.
Batoul Hojeij, Ilja Tchetverikov, Marc R. Kok, Marijn Vis, Yvonne Goekoop‐Ruiterman, Jessica Bijsterbosch, Paul Baudoin, Reinhard Bos, Jos H. van der Kaap, Petra Kok, Lindy‐Anne Korswagen, Jolanda J. Luime   +11 more
wiley   +1 more source

A Return to Normality: A Descriptive Qualitative Interview Study Exploring the Patient Experience of Gout Flare Resolution

Arthritis Care &Research, EarlyView.
Objective Although the definition of a gout flare is well established, the state of gout flare resolution has not yet been defined. This study aimed to explore patients’ experiences and perceptions of gout flare resolution. Methods Semistructured interviews were conducted with 24 people with gout, guided by open‐ended questions exploring their ...
Sarah Stewart, Anne Horne, Angelo Gaffo, Lisa K. Stamp, N. Lawrence Edwards, Nicola Dalbeth   +5 more
wiley   +1 more source

β‐Catenin/c‐Myc Axis Modulates Autophagy Response to Different Ammonia Concentrations

Advanced Biology, Volume 9, Issue 3, March 2025.
Ammonia, detoxified by the liver into urea and glutamine, impacts autophagy differently at varying levels. Low ammonia activates autophagy via c‐Myc and β‐catenin, while high levels suppress it. Using Huh7 cells and Spf‐ash mice, c‐Myc's role in cytoprotective autophagy is revealed, offering insights into hyperammonemia and potential therapeutic ...
S. Sergio, B. Spedicato, G. Corallo, A. Inguscio, M. Greco, D. Musarò, D. Vergara, A. F. Muro, G. De Sabbata, L. R. Soria, N. Brunetti Pierri, M. Maffia   +11 more
wiley   +1 more source

Hydrogen Occupancy, Site Hierarchy, and Hydride-Transformation Pathways in BCC High-Entropy Alloys. [PDF]

Molecules
Chen C, Hou Q, Gao L, Ding Z.
europepmc   +1 more source

Variable Stiffness Flexure Structures Enabled by Phase‐Change Gallium and Adhesive Interfacial Locking for Soft Robotic Applications

Advanced Engineering Materials, EarlyView.
A flexure‐based variable stiffness structure is developed by integrating phase‐change modulation and adhesive interfacial locking of gallium. The design enables a wide stiffness variability, transitioning from soft, flexible behavior to rigid, load‐supporting performance.
Sungjin Kim, Sohee John Yoon, Yong‐Lae Park   +2 more
wiley   +1 more source