Results 41 to 50 of about 68,097 (255)

Onasemnogene Abeparvovec in Type I Spinal Muscular Atrophy: 24‐Month Follow‐Up From the Italian Registry

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Onasemnogene abeparvovec (OA) is an AAV9‐based gene therapy for spinal muscular atrophy type I (SMA I). Real‐world outcomes show increased response variability compared to clinical trials, and follow‐up data beyond 12–18 months are limited.
Marika Pane, Giorgia Coratti, Chiara Cutrì, Antonio Varone, Riccardo Masson, Adele D'Amico, Valeria Sansone, Sonia Messina, Federica Ricci, Chiara Ticci, Claudio Bruno, Caterina Agosto, Francesca Benedetti, Antonella Pini, Sabrina Siliquini, Massimiliano Filosto, Alberto Zambon, Ilaria Bitetti, Maria Rosaria Manna, Claudia Dosi, Riccardo Zanin, Stefano Parravicini, Roberto De Sanctis, Giulia Stanca, Michela Catteruccia, Michele Tosi, Irene Mizzoni, Emilio Albamonte, Valentina Franchino, Maria Sframeli, Ilaria Cavallina, Elena Procopio, Michele Sacchini, Simone Morando, Noemi Brolatti, Federica Trucco, Gaia Scarpini, Elena Briganti, Beatrice Berti, Concetta Palermo, Daniela Leone, Stefano C. Previtali, Eugenio Mercuri, the ITASMAC working group   +43 more
wiley   +1 more source

Myoblast models of skeletal muscle hypertrophy and atrophy [PDF]

Current Opinion in Clinical Nutrition and Metabolic Care, 2011
To highlight recent breakthroughs and controversies in the use of myoblast models to uncover cellular and molecular mechanisms regulating skeletal muscle hypertrophy and atrophy.Myoblast cultures provide key mechanistic models of the signalling and molecular pathways potentially employed by skeletal muscle in-vivo to regulate hypertrophy and atrophy ...
Sharples, Adam P., Stewart, Claire E.
openaire   +3 more sources

Early Clinical, Imaging, and Pathological Characteristics of SRPK3/TTN‐Digenic Myopathy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective SRPK3/TTN‐digenic myopathy was recently established as a skeletal muscle myopathy caused by digenic inheritance. This study characterizes the early clinical presentation of SRPK3/TTN‐digenic myopathy in one previously reported and seven newly identified pediatric patients.
Rotem Orbach, Sandra Donkervoort, Carola Hedberg‐Oldfors, Giovanni Baranello, Dimah Saade, Precilla D'Souza, Ruchee Patel, Eva Michael, A. Reghan Foley, Diana Bharucha‐Goebel, S. Jin Haugland, Meghan McAnally, Omer Abdul Hamid, Katherine Chao, Ellen F. Macnamara, Alan H. Beggs, Anna Sarkozy, Juliane Mueller, Steven A. Moore, Richard S. Finkel, Cynthia J. Tifft, Francesco Muntoni, Anders Oldfors, Carsten G. Bönnemann   +23 more
wiley   +1 more source

Plasma EV Proteomics Identifies ECM Remodeling and Inflammatory Proteins LUM and C7 as Candidate Biomarkers in FSHD

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Facioscapulohumeral muscular dystrophy (FSHD) is one of the most debilitating and common muscular dystrophies. Despite its severity, no approved therapy exists for FSHD patients. However, several therapeutic candidates are currently under development, and some have recently entered clinical trials, marking the need for reliable ...
Mustafa Bilal Bayazit, Chiranth K. Nagaraj, Jackson S. Newell, Kim Truc Nguyen, Xilal Y. Rima, Jacob Doon‐Ralls, Eduardo Reátegui, Jeffrey M. Statland, Rabi Tawil, Kevin M. Flanigan, Scott Q. Harper, Nizar Y. Saad   +11 more
wiley   +1 more source

Obesity Subtypes and Longitudinal Trajectories of Function Over Seven Years of Follow‐Up: Data From the Multicenter Osteoarthritis Study

Arthritis Care &Research, EarlyView.
Objective Obesity, defined by body mass index (BMI) ≥30 kg/m2, is a risk factor for functional limitations in people with knee osteoarthritis (OA). However, function varies among such individuals. Our objective was to evaluate the implications of obesity subtypes on longitudinal patterns of physical functioning in people with or at risk for knee OA ...
Kristine Godziuk, Sarah Tilley, Michael LaValley, Michael C. Nevitt, Cora E. Lewis, James C. Torner, Tuhina Neogi, for the Multicenter Osteoarthritis Study Group   +7 more
wiley   +1 more source

3D Printing Innovations in Polymeric Porous and Patterned Architecture

Advanced Functional Materials, EarlyView.
Polymeric foams occupy a unique structural space between dense solids and open networks, where engineered void fraction governs mechanical compliance, thermal resistance, and mass transport. Additive manufacturing now enables precise spatial control over cellular architecture, unlocking designer foam structures across applications spanning crash ...
Dhanush Patil, Sri Vaishnavi Thummalapalli, Clarissa Westover, Arunachalam Ramanathan, M. Taylor Sobzcak, Roumika Patil, Qixuan Xiang, Tiantian Li, Sui Yang, Timothy E. Long, Hongyue Sun, Xianqiao Wang, Yang Liu, Kenan Song   +13 more
wiley   +1 more source

Blood Biomarkers and Surface‐Enhanced Raman Spectroscopy for Gout: A Comprehensive Review

Advanced Functional Materials, EarlyView.
Schematic illustrating gout disease progression from asymptomatic hyperuricemia to chronic tophaceous disease, highlighting the limitations of conventional imaging and biochemical diagnostics and the potential of engineered SERS platforms for ultrasensitive blood‐based detection of urate‐related biomarkers across disease stages, with the color gradient
Isuri Perera, Chiranjeevi Kanike, Appurva Tiwari, Hongyan Wu, Larry D. Unsworth, Ben Bin Xu, Xuehua Zhang   +6 more
wiley   +1 more source

Effects and mechanisms of phytochemicals on skeletal muscle atrophy in glucolipid metabolic disorders: current evidence and future perspectives

Food Innovation and Advances
Skeletal muscle atrophy resulting from glucolipid metabolic disorders poses a serious challenge to human health with the rapidly increasing prevalence of diabetes and obesity.
Mengjie Li, Yige Qin, Ruixuan Geng, Jingjing Fang, Seong-Gook Kang, Kunlun Huang, Tao Tong   +6 more
doaj   +1 more source

Targeting Post‐Irradiation Thyroid Dysfunction: Electrospun Scaffolds As A Dual‐Action Approach for Antioxidant and Immune Modulation

Advanced Healthcare Materials, EarlyView.
Radiation‐induced hypothyroidism follows head and neck radiotherapy due to oxidative stress and inflammation. Electrospun polycaprolactone scaffolds containing adenosine have potential to modulate thyroid repair. Scaffolds enhance thyrocyte proliferation, antioxidant enzymes glutathione peroxidase and catalase, reduce senescence and apoptosis markers ...
Maria Heim, Ella‐Louise Handley, Daniel Grant, Lizi M. Hegarty, Elaine Emmerson, Anthony Callanan   +5 more
wiley   +1 more source

Resistance exercise exacerbates muscle atrophy of skeletal muscle-specific Beclin1 conditioned knockout (cKO) mice

Advanced Exercise and Health Science
The autophagy-lysosome pathway is the major route for protein and organelle clearance, and the functional status of autophagy plays an important role in the performance of skeletal muscle.
Zhengzhong Zeng, Hu Zhang, Jiling Liang, Jun Lv, Liangwen Wu, Ning Chen   +5 more
doaj   +1 more source