Results 71 to 80 of about 142,613 (313)

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito, Kamal Benguerba, Ken Tsuchida, Kazushige Yazawa, Isao Tsumiyama, Hiromitsu Kayama, Sandra P. Reyna, Farid Khan, Richard S. Finkel   +8 more
wiley   +1 more source

Convergence of MTOR and Glucocorticoid receptor signalling in the human placenta: effects of pre-term labour, nutrition and maternal stress [PDF]

, 2011
This thesis was submitted for the degree of Doctor of Philosophy and awarded by Brunel University.A vital factor for foetal development is the nutrient transport at placental level.
Mparmpakas, Dionisis G.
core  

A Qualitative Analysis of Patient Perspectives and Preferences in Lupus Management to Guide Lupus Guidelines Development

Arthritis Care &Research, EarlyView.
Objective A patient‐centered approach for chronic disease management, including systemic lupus erythematosus (SLE), aligns treatment with patients’ values and preferences, leading to improved outcomes. This paper summarizes how patient experiences, perspectives, and priorities informed the American College of Rheumatology (ACR) 2024 Lupus Nephritis (LN)
Shivani Garg, Izzy Hartel, Lisa R. Sammaritano, Anca Askanase, Bonnie L. Bermas, Maria Dall'Era, Alí Duarte‐García, Victoria P. Werth, Brad Rovin, Reem A. Mustafa, Amy S. Turner, Bené Williams, Brian L. Ung, Hiya Bhavsar, Monique C. Gore‐Massy, Nahirannette Pulido, Natacha Guerrero, Natalie M. Smith, Wambui Machua, Linda T. Hiraki, Mary Beth Son   +20 more
wiley   +1 more source

Inhibiting glucocorticoid receptors enhances adult spinal cord neural stem cell activity and improves outcomes in spinal cord injury

Communications Biology
The spinal cord injury (SCI) microenvironment undergoes significant biochemical changes that influence the behavior of endogenous neural stem/progenitor cells (NSPCs), including elevated glucocorticoid levels.
Xuefeng Zhang, Siyuan Zhou, Shengyu Tang, Xiaotong Hou, Yonghui Cai, Changlong Hu   +5 more
doaj   +1 more source

Nonclassic lipoid congenital adrenal hyperplasia masquerading as familial glucocorticoid deficiency

, 2009
Context: Familial glucocorticoid deficiency (FGD) is an autosomal recessive disorder resulting from resistance to the action of ACTH on the adrenal cortex.
Racine, M, Naville, D., Nurnberg, G., Green, J, Metherell, L. A., Nurnberg, P, Krone, NP, Krone, N. P., Arlt, W, Tomlinson, Jeremy, Achermann, JC, Halaby, G, Tomlinson, J. W., Clark, A. J. L., Krone, Nils, Naville, D, Berney, D. M., Begeot, M., Huebner, A., Halaby, G., Green, J., Berney, DM, Clark, AJ, Lin, L, Lin, L., Racine, M., Arlt, Wiebke, Achermann, J. C., Arlt, W., Nurnberg, G, Metherell, LA, Tomlinson, JW, Begeot, M, Huebner, A, Nurnberg, P., Clark, AJL   +35 more
core   +1 more source

Validation of the Pediatric Arthritis Ultrasound Scoring System for the Elbow, Wrist, and Finger Joints in Children With Juvenile Idiopathic Arthritis

Arthritis Care &Research, EarlyView.
Objective We aimed to validate the Pediatric Arthritis Ultrasound Scoring System (PAUSS) for upper extremity joints in children with juvenile idiopathic arthritis (JIA). Methods Children with JIA were evaluated for elbow, wrist, or finger arthritis by clinical examination (CE) and musculoskeletal ultrasound (MSUS) with images scored according to the ...
Patricia Vega‐Fernandez, Pinar Ozge Avar‐Aydin, Kelly Rogers, Daniel J. Lovell, Jennifer Huggins, Alexei Grom, Hermine I. Brunner, Grant S. Schulert, Sheila T. Angeles‐Han, Mekibib Altaye, Amy Cassedy, Arthur B. Meyers, Tracy V. Ting   +12 more
wiley   +1 more source

01 | OVERCOMING HORMONAL BARRIERS TO CARDIAC REGENERATION

European Journal of Histochemistry
Cardiomyocyte loss after acute or chronic myocardial injury is a major cause of heart failure. Adult mammalian cardiomyocytes retain a very limited proliferative capacity, insufficient for effective regeneration.
GEI - SOCIETÀ ITALIANA DI BIOLOGIA DELLO SVILUPPO E DELLA CELLULA
doaj   +1 more source

Glucocorticoid receptor gene polymorphisms do not affect growth in fetal and early postnatal life. The Generation R Study

BMC Medical Genetics, 2010
Background Glucocorticoids have an important role in early growth and development. Glucocorticoid receptor gene polymorphisms have been identified that contribute to the variability in glucocorticoid sensitivity.
Raat Hein, Moll Henriette A, van Rossum Elisabeth FC, Koper Jan W, Steegers Eric AP, Geelhoed Miranda JJ, Tiemeier Henning, Hofman Albert, Jaddoe Vincent WV   +8 more
doaj   +1 more source

Sex Differences in Medication Discontinuation in Axial Spondyloarthritis

Arthritis Care &Research, EarlyView.
Objective We examined sex differences in medication discontinuation among patients with axial spondyloarthritis (axSpA) initiating tumor necrosis factor inhibitors (TNFi), interleukin‐17 inhibitors (IL‐17i), or JAK inhibitors (JAKi). Methods Using data from the Rheumatology Informatics System for Effectiveness (RISE) Registry (2003–2025), we assessed ...
Rachael Stovall, Cinthia Blat, Eric Roberts, Jean W. Liew, Katherine Wysham, Namrata Singh, Janna Friedly, Lianne S. Gensler, Gabriela Schmajuk, Jinoos Yazdany   +9 more
wiley   +1 more source

Familial glucocorticoid receptor haploinsufficiency by non-sense mediated mRNA decay, adrenal hyperplasia and apparent mineralocorticoid excess. [PDF]

PLoS ONE, 2010
Primary glucocorticoid resistance (OMIM 138040) is a rare hereditary disease that causes a generalized partial insensitivity to glucocorticoid action, due to genetic alterations of the glucocorticoid receptor (GR). Investigation of adrenal incidentalomas
Jérôme Bouligand, Brigitte Delemer, Annie-Claude Hecart, Geri Meduri, Say Viengchareun, Larbi Amazit, Séverine Trabado, Bruno Fève, Anne Guiochon-Mantel, Jacques Young, Marc Lombès   +10 more
doaj   +1 more source