未登录
分享
收藏Performance of three simultaneously used rtCGM systems around a physically active weekend camp in adults with type 1 diabetes: A prospective lab and real-world study
Sanfilippo, Sabrina; Thurm, Ulrike; Renfordt, Mats; Schierbauer, Janis; Sourij, Harald; Zimmermann, Paul; Voit, Thomas; Tschakert, Gerhard; Kapan, Ali; Moser, Othmar
分享
收藏miR-379 links glucocorticoid treatment with mitochondrial response in Duchenne muscular dystrophy (vol 10, 9139, 2020)
Sanson, Mathilde; Vu Hong, Ai; Massourides, Emmanuelle; Bourg, Nathalie; Suel, Laurence; Amor, Fatima; Corre, Guillaume; Benit, Paule; Barthelemy, Ines; Blot, Stephane; Bigot, Anne; Pinset, Christian; Rustin, Pierre; Servais, Laurent; Voit, Thomas; Richard, Isabelle; Israeli, David
分享
收藏Impact of AAVMYBPC3 Gene Transfer on Heart Structure and Function in Human and Mouse Models of Hypertrophic Cardiomyopathy
Vitelli, Catherine; Zhang, Lening; Murphy, Ryan; Blus, Bartlomiej; Christianson, Terri; Giaramita, Alexander; Holtzinger, John; Handyside, Britta; May, Hannah; Ann Pham; Tu Nguyen; Zhou, Huiyu; Ntai, Ioanna; Mearini, Giulia; Ricotti, Valeria; Voit, Thomas; Eschenhagen, Thomas; Carrier, Lucie; Agarwal, Pooja; Bunting, Stuart; Sumandea, Marius
分享
收藏
分享
收藏A wearable motion capture suit and machine learning predict disease progression in Friedreich's ataxia
Kadirvelu, Balasundaram; Gavriel, Constantinos; Nageshwaran, Sathiji; Chan, Jackson Ping Kei; Nethisinghe, Suran; Athanasopoulos, Stavros; Ricotti, Valeria; Voit, Thomas; Giunti, Paola; Festenstein, Richard; Faisal, A. Aldo
分享
收藏
分享
收藏
分享
收藏Cholesterol metabolism is a potential therapeutic target in Duchenne muscular dystrophy
Amor, Fatima; Vu Hong, Ai; Corre, Guillaume; Sanson, Mathilde; Suel, Laurence; Blaie, Stephanie; Servais, Laurent; Voit, Thomas; Richard, Isabelle; Israeli, David
分享
收藏International retrospective natural history study of LMNA-related congenital muscular dystrophy
Ben Yaou, Rabah; Yun, Pomi; Dabaj, Ivana; Norato, Gina; Donkervoort, Sandra; Xiong, Hui; Nascimento, Andres; Maggi, Lorenzo; Sarkozy, Anna; Monges, Soledad; Bertoli, Marta; Komaki, Hirofumi; Mayer, Michele; Mercuri, Eugenio; Zanoteli, Edmar; Castiglioni, Claudia; Marini-Bettolo, Chiara; D'Amico, Adele; Deconinck, Nicolas; Desguerre, Isabelle; Erazo-Torricelli, Ricardo; Gurgel-Giannetti, Juliana; Ishiyama, Akihiko; Kleinsteuber, Karin S.; Lagrue, Emmanuelle; Laugel, Vincent; Mercier, Sandra; Messina, Sonia; Politano, Luisa; Ryan, Monique M.; Sabouraud, Pascal; Schara, Ulrike; Siciliano, Gabriele; Vercelli, Liliana; Voit, Thomas; Yoon, Grace; Alvarez, Rachel; Muntoni, Francesco; Pierson, Tyler M.; Gomez-Andres, David; Foley, A. Reghan; Quijano-Roy, Susana; Bonnemann, Carsten G.; Bonne, Gisele
分享
收藏
分享
收藏Novel free-circulating and extracellular vesicle-derived miRNAs dysregulated in Duchenne muscular dystrophy
Catapano, Francesco; Scaglioni, Dominic; Maresh, Kate; Ala, Pierpaolo; Domingos, Joana; Selby, Victoria; Ricotti, Valeria; Phillips, Lauren; Servais, Laurent; Seferian, Andreea; de Groot, Imelda; Krom, Yvonne D.; Voit, Thomas; Verschuuren, J. J. G. M.; Niks, E. H.; Straub, Volker; Morgan, Jennifer; Muntoni, Francesco
分享
收藏GGPS1Mutations Cause Muscular Dystrophy/Hearing Loss/Ovarian Insufficiency Syndrome
Foley, A. Reghan; Zou, Yaqun; Dunford, James E.; Rooney, Jachinta; Chandra, Goutam; Xiong, Hui; Straub, Volker; Voit, Thomas; Romero, Norma; Donkervoort, Sandra; Hu, Ying; Markello, Thomas; Horn, Adam; Qebibo, Leila; Dastgir, Jahannaz; Meilleur, Katherine G.; Finkel, Richard S.; Fan, Yanbin; Mamchaoui, Kamel; Duguez, Stephanie; Nelson, Isabelle; Laporte, Jocelyn; Santi, Mariarita; Malfatti, Edoardo; Maisonobe, Thierry; Touraine, Philippe; Hirano, Michio; Hughes, Imelda; Bushby, Kate; Oppermann, Udo; Bohm, Johann; Jaiswal, Jyoti K.; Stojkovic, Tanya; Bonnemann, Carsten G.
分享
收藏
分享
收藏
分享
收藏Downregulation of miRNA-29,-23 and-21 in urine of Duchenne muscular dystrophy patients
Catapano, Francesco; Domingos, Joana; Perry, Mark; Ricotti, Valeria; Phillips, Lauren; Servais, Laurent; Seferian, Andreea; de Groot, Imelda; Krom, Yvonne D.; Niks, Erik H.; Verschuuren, Jan J. G. M.; Straub, Volker; Voit, Thomas; Morgan, Jennifer; Muntoni, Francesco
分享
收藏RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect
Domenger, Claire; Allais, Marine; Francois, Virginie; Leger, Adrien; Lecomte, Emilie; Montus, Marie; Servais, Laurent; Voit, Thomas; Moullier, Philippe; Audic, Yann; Le Guiner, Caroline
分享
收藏OUTCOME MEASURES FOR DUCHENNE MUSCULAR DYSTROPHY: IMPLICATIONS FOR TRIALS
Domingos, Joana; Eagle, Michelle; Moraux, Amelie; Butler, Jordan; Decostre, Valerie; Ridout, Deborah; Mayhew, Anna; Selby, Victoria; Guglieri, Michela; Van der Holst, Menno; Jansen, Merel; Verschuuren, Jan G. M.; de Groot, Imelda; Niks, Erik; Servais, Laurent; Hogrel, Jean-Yves; Straub, Volker; Voit, Thomas; Ricotti, Valeria; Muntoni, Francesco
分享
收藏Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
Mariot, Virginie; Joubert, Romain; Hourde, Christophe; Feasson, Leonard; Hanna, Michael; Muntoni, Francesco; Maisonobe, Thierry; Servais, Laurent; Bogni, Caroline; Le Panse, Rozen; Benvensite, Olivier; Stojkovic, Tanya; Machado, Pedro M.; Voit, Thomas; Buj-Bello, Ana; Dumonceaux, Julie
分享
收藏A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model
Biferi, Maria Grazia; Cohen-Tannoudji, Mathilde; Cappelletto, Ambra; Giroux, Benoit; Roda, Marianne; Astord, Stephanie; Marais, Thibaut; Bos, Corinne; Voit, Thomas; Ferry, Arnaud; Barkats, Martine
分享
收藏