Search Results - "Duchenne muscular dystrophy"

Aldehyde dehydrogenases contribute to skeletal muscle homeostasis in healthy, aging, and Duchenne muscular dystrophy patients by Jessy Etienne, Pierre Joanne, Cyril Catelain, Stéphanie Riveron, Alexandra Clarissa Bayer, Jérémy Lafable, Isabel Punzon, Stéphane Blot, Onnik Agbulut, Jean‐Thomas Vilquin

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Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy by Shin'ichi Takeda, Takashi Okada

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Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington's Disease by Ariana P. Mullin, Diane Corey, Emily C. Turner, Richard Liwski, Daniel Olson, Jackson Burton, Sudhir Sivakumaran, Lynn D. Hudson, Klaus Romero, Diane T. Stephenson, Jane Larkindale

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Siblings' life aspirations in the context of Duchenne muscular dystrophy: a mixed-methods case-control study by Carolyn E. Schwartz, Elijah Biletch, Richard B. B. Stuart, Bruce D. Rapkin

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Patient demographics and characteristics from an ambispective, observational study of patients with duchenne muscular dystrophy in Saudi Arabia by Abdulaziz S. AlSaman, Fouad Al Ghamdi, Ahmed K. Bamaga, Ahmed K. Bamaga, Nahla AlShaikh, Nahla AlShaikh, Nahla AlShaikh, Mohammed Al Muqbil, Mohammed Al Muqbil, Mohammed Al Muqbil, Osama Muthaffar, Osama Muthaffar, Fahad A. Bashiri, Baleegh Ali, Arzu Mulayim, Elena Heider, Abdullah A. Alshahrani, Mohammed A. Al Muhaizea

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Multivariate modeling of magnetic resonance biomarkers and clinical outcome measures for Duchenne muscular dystrophy clinical trials by Sarah Kim, Rebecca J. Willcocks, Michael J. Daniels, Juan Francisco Morales, Deok Yong Yoon, William T. Triplett, Alison M. Barnard, Daniela J. Conrado, Varun Aggarwal, Ramona Belfiore‐Oshan, Terina N. Martinez, Glenn A. Walter, William D. Rooney, Krista Vandenborne

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Functional cardiac consequences of β-adrenergic stress-induced injury in a model of Duchenne muscular dystrophy by Conner C. Earl, Areli J. Javier, Alyssa M. Richards, Larry W. Markham, Craig J. Goergen, Steven S. Welc

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Can simple and low-cost motor function assessments help in the diagnostic suspicion of Duchenne muscular dystrophy? by Aline Chacon Pereira, Alexandra Prufer de Queiroz Campos Araújo, Márcia Gonçalves Ribeiro

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Correction of human nonsense mutation via adenine base editing for Duchenne muscular dystrophy treatment in mouse by Ming Jin, Jiajia Lin, Haisen Li, Zhifang Li, Dong Yang, Yin Wang, Yuyang Yu, Zhurui Shao, Long Chen, Zhiqiang Wang, Yu Zhang, Xiumei Zhang, Ning Wang, Chunlong Xu, Hui Yang, Wan-Jin Chen, Guoling Li

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Consensus Statement on the Management of Duchenne Muscular Dystrophy in Saudi Arabia During the Coronavirus Disease 2019 Pandemic by Ahmed K. Bamaga, Ahmed K. Bamaga, Fouad Alghamdi, Nahla Alshaikh, Nahla Alshaikh, Nahla Alshaikh, Waleed Altwaijri, Waleed Altwaijri, Fahad A. Bashiri, Fahad A. Bashiri, Khalid Hundallah, Musaad Abukhaled, Osama Y. Muthaffar, Osama Y. Muthaffar, Sameer Al-Mehmadi, Tahani Ahmed Jamaly, Mohammad A. Al-Muhaizea, Abdulaziz Al-Saman

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Can simple and low-cost motor function assessments help in the diagnostic suspicion of Duchenne muscular dystrophy? by Aline Chacon Pereira, Alexandra Prufer de Queiroz Campos Araújo, Márcia Gonçalves Ribeiro

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Walking alone milestone combined reading-frame rule improves early prediction of Duchenne muscular dystrophy by Yan-li Ma, Yan-li Ma, Wei-hua Zhang, Guo-hong Chen, Li-fang Song, Yuan Wang, Rui-li Yuan, Ying Wang, Xiu-yong Cheng

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Unveiling the Respiratory Muscle Strength in Duchenne Muscular Dystrophy: The Impact of Nutrition and Thoracic Deformities, Beyond Spirometry by Mine Yuksel Kalyoncu, Yasemin Gokdemir, Cansu Yilmaz Yegit, Muruvvet Yanaz, Aynur Gulieva, Merve Selcuk, Şeyda Karabulut, Neval Metin Çakar, Pinar Ergenekon, Ela Erdem Eralp, Gülten Öztürk, Olcay Unver, Dilsad Turkdogan, Yavuz Sahbat, Ahmet Hamdi Akgülle, Fazilet Karakoç, Bulent Karadag

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Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status by Craig M McDonald, Jessica R Marden, Perry B Shieh, Brenda L Wong, Henry Lane, Adina Zhang, Ha Nguyen, Molly Frean, Panayiota Trifillis, Karyn Koladicz, James Signorovitch

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Interplay of disability, caregiver impact, and out-of-pocket expenditures in Duchenne muscular dystrophy: a cohort study by Carolyn E. Schwartz, Roland B. Stark, Katrina Borowiec, Ivana F. Audhya, Katherine L. Gooch

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Duchenne Muscular Dystrophy With Low Acidic α-Glucosidase Activity: Two Case Reports and Literature Review by Xiufang He, Xiufang He, Xuandi Li, Xuandi Li, Yuese Lin, Yuese Lin, Hongjun Ba, Hongjun Ba, Huimin Peng, Huimin Peng, Lili Zhang, Lili Zhang, Ling Zhu, Ling Zhu, Youzhen Qin, Youzhen Qin, Shujuan Li, Shujuan Li

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Patient life aspirations in the context of Duchenne Muscular Dystrophy: a mixed-methods case-control study by Carolyn E. Schwartz, Elijah Biletch, Richard B. B. Stuart, Bruce D. Rapkin

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Serum inflammatory cytokines as disease biomarkers in the DE50-MD dog model of Duchenne muscular dystrophy by Dominique O. Riddell, John C. W. Hildyard, Rachel C. M. Harron, Natasha L. Hornby, Dominic J. Wells, Richard J. Piercy

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In Vitro Studies to Evaluate the Intestinal Permeation of an Ursodeoxycholic Acid-Conjugated Oligonucleotide for Duchenne Muscular Dystrophy Treatment by Marika Faiella, Giada Botti, Alessandro Dalpiaz, Lorenzo Gnudi, Aurélie Goyenvalle, Barbara Pavan, Daniela Perrone, Matteo Bovolenta, Elena Marchesi

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Noninvasive prenatal diagnosis of duchenne muscular dystrophy in five Chinese families based on relative mutation dosage approach by Ganye Zhao, Xiaofeng Wang, Lina Liu, Peng Dai, Xiangdong Kong

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