Results 71 to 80 of about 72,020 (228)

Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes

Pediatrics, 2020
In this article, we discuss overall safety and early outcomes in a large cohort of patients with SMA treated with onasemnogene abeparvovec-xioi within Ohio.
M. Waldrop, Cassandra Karingada, Michael A Storey, B. Powers, M. Iammarino, N. Miller, L. Alfano, G. Noritz, Ian T. Rossman, M. Ginsberg, Kathryn A. Mosher, E. Broomall, Jessica Goldstein, N. Bass, L. Lowes, C. Tsao, J. Mendell, A. Connolly   +17 more
semanticscholar   +1 more source

Development of a Vaginal Extracellular Matrix Hydrogel for Combating Genitourinary Syndrome of Menopause

Advanced Materials, EarlyView.
In this study, a vaginal specific ECM hydrogel (vECM) is developed, optimized for intravaginal topical administration, and evaluated for mitigatation of vaginal atrophy associated with menopause. When applied in a rat model of menopause, vECM improves vaginal epithelial thickness and stem cell phenotype, and despite topical application, vECM also ...
Emma I. Zelus, Jacqueline Grime, Anthony Saviola, Maxwell McCabe, Kirk C. Hansen, Marianna Alperin, Karen L. Christman   +6 more
wiley   +1 more source

Natural history of Type 2 and 3 spinal muscular atrophy: 2‐year NatHis‐SMA study

Annals of Clinical and Translational Neurology, 2020
To characterize the natural history of spinal muscular atrophy (SMA) over 24 months using innovative measures such as wearable devices, and to provide evidence for the sensitivity of these measures to determine their suitability as endpoints in clinical ...
M. Annoussamy, A. Seferian, A. Daron, Y. Péréon, C. Cances, C. Vuillerot, Liesbeth De Waele, V. Laugel, U. Schara, T. Gidaro, C. Lilien, J. Hogrel, P. Carlier, E. Fournier, L. Lowes, K. Gorni, Myriam Ly-Le Moal, N. Hellbach, T. Seabrook, C. Czech, R. Hermosilla, L. Servais   +21 more
semanticscholar   +1 more source

Defective axonal transport in motor neuron disease [PDF]

, 2007
Several recent studies have highlighted the role of axonal transport in the pathogenesis of motor neuron diseases. Mutations in genes that control microtubule regulation and dynamics have been shown to cause motor neuron degeneration in mice and in a ...
Baas, Blair, Boillee, Bommel, Borchelt, Bowling, Brown, Bruijn, Burger, Cao, Cleveland, Cleveland, Duchen, Escurat, Evans, Fichera, Fliegner, Gaudette, Goldstein, Hadano, Hafezparast, Hazan, Hirokawa, Hirokawa, Hirokawa, Jablonka, Joshi, Kanai, Kaplan, Kieran, King, Kong, LaMonte, Levy, Ligon, Liu, Lo Giudice, Lu, Martin, Miki, Miki, Mitsumoto, Mitsumoto, Muglia, Munch, Nicholson, Nishimura, Okabe, Otomo, Parkinson, Parysek, Pasinelli, Patel, Proukakis, Puls, Puls, Quenneville, Rao, Reid, Reid, Reid, Rosen, Ross, Rowland, Sanderson, Schmitt-John, Shaw, Shibata, Siegel, Song, Teuchert, Troy, Vallee, Weber, Williamson, Wood, Xia, Zhao   +77 more
core   +1 more source

Fabrication of Organic/Inorganic Nanocomposites: From Traditional Synthesis to Additive Manufacturing

Advanced Materials, EarlyView.
It provides an overview of the synthesis of organic/inorganic nanocomposites, spanning from conventional methods to advanced 3D printing techniques. It summarizes key challenges in 3D‐printed nanocomposites and highlights emerging innovations, including 5D printing, AI‐assisted material design, nanoscale additive manufacturing, and sustainable closed ...
Liwen Zhang, Xumin Huang, Liwei Liu, Naufal Kabir Ahamed Nasar, Xinyan Gu, Thomas P. Davis, Xiaoyu (Rayne) Zheng, Lianzhou Wang, Ruirui Qiao   +8 more
wiley   +1 more source

Natural history of lung function in spinal muscular atrophy

Orphanet Journal of Rare Diseases, 2020
Background Respiratory muscle weakness is an important feature of spinal muscular atrophy (SMA). Progressive lung function decline is the most important cause of mortality and morbidity in patients.
C. Wijngaarde, E. Veldhoen, R. V. van Eijk, M. Stam, L. Otto, F. Asselman, R. W. Wösten-van Asperen, E. Hulzebos, Laura P. Verweij-van den Oudenrijn, B. Bartels, I. Cuppen, R. Wadman, Leonard H. van den Berg, C. K. van der Ent, W. L. van der Pol   +14 more
semanticscholar   +1 more source

3D Printing for Neural Repair: Bridging the Gap in Regenerative Medicine

Advanced Materials, EarlyView.
This perspective article discusses how 3D bioprinting is advancing the development of neural tissue models and implants. It highlights recent progress in fabricating complex, multicellular neural constructs, examines current technical barriers, and outlines future applications in disease modeling, neurotoxicity testing, and regenerative therapies ...
Mitchell St Clair‐Glover, Zhilian Yue, Mirella Dottori   +2 more
wiley   +1 more source

Tremor in motor neuron disease may be central rather than peripheral in origin [PDF]

, 2018
BACKGROUND AND PURPOSE: Motor neuron disease (MND) refers to a spectrum of degenerative diseases affecting motor neurons. Recent clinical and post-mortem observations have revealed considerable variability in the phenotype.
Balint, B, Batla, A, Berardelli, A, Bhatia, K P, Ciocca, M, Latorre, A, Rocchi, L, Rothwell, J C, Sidle, K, Stamelou, M   +9 more
core   +2 more sources

Lightweight and High‐Performance Ankle Braces Enabled by 3D Printed Metamaterial Liquid Crystal Polymer

Advanced Materials Technologies, EarlyView.
A flexible, lightweight ankle brace made from 3D‐printed liquid crystal polymer metamaterials offers targeted support without restricting movement. Programmable stiffness provides protection during risky motions. Biomechanical tests suggest performance comparable to commercial braces with reduced bulk, highlighting potential for broader orthotic ...
Cassi J. Henderson, Zehao Ji, Miaomiao Zou, Xijin Hua, Thomas Stone, Sebastian W. Pattinson   +5 more
wiley   +1 more source

Rewiring Neuroimmunity: Nanoplatform Innovations for CNS Disease Therapy

Advanced Therapeutics, EarlyView.
This review explores emerging nanoplatform strategies designed to modulate neuroimmune responses for treating central nervous system (CNS) disorders. It examines structural and microenvironmental barriers, advances in multifunctional and targeted nanotechnologies, and highlights clinical progress and translational challenges, offering insights into the
Muhammad Usman Akbar, Weiwei Guo, Xin Shen, Yimeng Fang, Yilin Liu, Peng‐Yuan Wang, Roey Elnathan, Yaping Chen   +7 more
wiley   +1 more source