Results 51 to 60 of about 43,207 (322)

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

Moschcowitz Disease in pediatric. [PDF]

Batna Journal of Medical Sciences
Hemichorea Moskowitz disease is a rare hematological disorder known to be an autoimmune disease in most patients. Most cases of thrombotic thrombopenic purpura (TTP) are caused by problems with an enzyme or protein in the blood called ADAMTS13.
Samira AGGOUNE, Mohamed Faouzi SEDDIKI
doaj   +1 more source

Endothelial Cell Proteins as Biomarkers in Susac Syndrome

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Susac syndrome (SS) is a rare CD8+ T cell–mediated microangiopathy affecting the brain, retina, and auditory labyrinth. Endothelial injury is thought to be a central mechanism; however, no circulating disease biomarkers are known. We performed targeted proteomic profiling to identify circulating endothelial‐associated proteins as ...
Rohit Benjamin, Yue Andy Qi, Ying Hao, Catherine DeMarino, Samuel Darko, Irene Cortese, Daniel S. Reich, María I. Gaitán, Steven Jacobson, Bridgett J. Billioux, Bryan Smith, Avindra Nath   +11 more
wiley   +1 more source

Stasis microangiopathy: from pathogenesis to treatment [PDF]

, 2021
Stasis microangiopathy includes all the pathological changes in the microcirculation and interstitium due to venous hypertension. Venous valve incompetence occurring in the superficial or deep venous system or in both is the most common cause of venous ...
Bilancini, Salvino, Lucchi, Massimo, Ciacciarelli, Marco   +2 more
core  

The Lupus Damage Index Revision Program: Results From the Item Generation and Reduction Phases

Arthritis Care &Research, EarlyView.
Objective A data‐driven and expert/patient consensus‐based project to develop a revised Systemic Lupus International Collaborating Clinics (SLICC)/American College of Rheumatology (ACR) Damage Index (SDI) is under way supported by SLICC, ACR, and the Lupus Foundation of America. Our objective is to report the item generation and reduction phase results
Burak Kundakci, Megan R. W. Barber, Ann E. Clarke, Sindhu R. Johnson, Anselm Mak, Hermine I. Brunner, Murat Inanc, Rosalind Ramsey‐Goldman, Guillermo Ruiz‐Irastorza, Ellen M. Ginzler, John G. Hanly, Alexandra Legge, Cynthia Aranow, Laurent Arnaud, Nathalie Costedoat‐Chalumeau, Dafna D. Gladman, Søren Jacobsen, Michelle Petri, Clovis A. Silva, Évelyne Vinet, Alexandre E. Voskuyl, SDI Revision Literature Review Groups, SDI Revision Delphi Panel, SDI Revision Item Reduction Committee, SDI Revision Clinical Domain Groups, Ian N. Bruce   +25 more
wiley   +1 more source

Renal thrombotic microangiopathy caused by interferon beta-1a treatment for multiple sclerosis [PDF]

, 2013
Julien Mahe,1 Aurélie Meurette,2 Anne Moreau,3 Caroline Vercel,2 Pascale Jolliet1,4 1Clinical Pharmacology Department, Institute of Biology, University Hospital, Nantes, France; 2Clinical Nephrology and Immunology Department, University Hospital ...
Mahe J, Moreau A, Jolliet P, Meurette A, Vercel C   +4 more
core  

Treatment of comorbid patients with chronic brain ischemia and vertebrogenic pain syndromes

Медицинский совет, 2019
Chronic brain ischemia and chronic pain both appear to be an extremely wide-spread disease. The reasons of widespread comorbidity of ischemic brain disease and chronic pain are common risk factors: old age, decrease of physical activity, syndrome of ...
V. V. Zakharov, V. V. Grinyuk, N. V. Vakhnina, E. Yu. Kalimeeva   +3 more
doaj   +1 more source

Vascularization of Human iPSC‐Derived Kidney Organoids Using Perfusion Culture, Pre‐Vascularized Collagen Scaffolds, and Decellularized Extracellular Matrix

Advanced Healthcare Materials, EarlyView.
An integrated perfusion‐based culture platform combining pre‐vascularized collagen scaffolds, stromal vascular fraction cells, and kidney decellularized extracellular matrix promotes vascularization of human iPSC‐derived kidney organoids. The platform provides evidence of interactions between vascular networks and glomerular‐like structures and enables
Helen Kearney, Andrea Mazzoleni, Ivan Martin, Lorenzo Moroni, Manuele Giuseppe Muraro, Carlos Mota   +5 more
wiley   +1 more source

Modulating Calcium Homeostasis via a Biomimetic Scaffold to Rescue Diabetic Ischemic Wounds

Advanced Science, EarlyView.
This strategy addresses impaired microcirculation and loss of extracellular matrix (ECM) guidance in diabetic wound healing. Musc@CP, a nanofibrous dressing combining an ECM‐mimetic chitosan‐pullulan scaffold with muscone, enhances perfusion by attenuating intracellular Ca2+ overload‐associated endothelial dysfunction.
Xiang Zheng, Mingmei Li, Jianjun Jiang, Yongchao Wang, Yaru Jia, Weilun Sun, Yi Yao, Pengli Gao, Linhua Zhang, Qi Guo, Guang Jia, Xing‐Jie Liang, Dunwan Zhu, Jinchao Zhang, Fangzhou Li   +14 more
wiley   +1 more source

POEMS Syndrome: 2026 Update on Diagnosis, Risk‐Stratification, and Management

American Journal of Hematology, EarlyView.
ABSTRACT Disease Overview POEMS syndrome is a life‐threatening syndrome due to an underlying plasma cell neoplasm. The major criteria for the syndrome are polyneuropathy, clonal plasma cell disorder (PCD), sclerotic bone lesions, elevated vascular endothelial growth factor, and the presence of Castleman disease.
Angela Dispenzieri
wiley   +1 more source