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Anzahl der Publikationen: 3
Zeitschriftenartikel
Dowling, James J.; Mueller-Felber, Wolfgang; Smith, Barbara K.; Bonnemann, Carsten G.; Kuntz, Nancy L.; Muntoni, Francesco; Servais, Laurent; Alfano, Lindsay N.; Beggs, Alan H.; Bilder, Deborah A.; Blaschek, Astrid; Duong, Tina; Graham, Robert J.; Jain, Minal; Lawlor, Michael W.; Lee, Jun; Coats, Julie; Lilien, Charlotte; Lowes, Linda P.; MacBean, Victoria; Neuhaus, Sarah; Noursalehi, Mojtaba; Pitts, Teresa; Finlay, Caroline; Christensen, Sarah; Rafferty, Gerrard; Seferian, Andreea M.; Tsuchiya, Etsuko; James, Emma S.; Miller, Weston; Sepulveda, Bryan; Vila, Maria Candida; Prasad, Suyash; Rico, Salvador und Shieh, Perry B.
(2022):
INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.
In: Journal of Neuromuscular Diseases, Bd. 9, Nr. 4: S. 503-516
Duong, Tina; Harding, Gale; Mannix, Sally; Abel, Cristina; Phillips, Dawn; Alfano, Lindsay N.; Bonnemann, Carsten G.; Lilien, Charlotte; Lowes, Linda P.; Servais, Laurent; Warken-Madelung, Birgit; Bergman, Susie Nieto; James, Emma S.; Noursalehi, Mojtaba; Prasad, Suyash; Rico, Salvador und Bilder, Deborah A.
(2021):
Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance.
In: Journal of Neuromuscular Diseases, Bd. 8, Nr. 1: S. 63-77
Jacobs, Marni B.; James, Meredith K.; Lowes, Linda P.; Alfano, Lindsay N.; Eagle, Michelle; Muni Lofra, Robert; Moore, Ursula; Feng, Jia; Rufibach, Laura E.; Rose, Kristy; Duong, Tina; Bello, Luca; Pedrosa-Hernandez, Irene; Holsten, Scott; Sakamoto, Chikako; Canal, Aurelie; Sanchez-Aguilera Praxedes, Nieves; Thiele, Simone; Siener, Catherine; Vandevelde, Bruno; DeWolf, Brittney; Maron, Elke; Guglieri, Michela; Hogrel, Jean-Yves; Blamire, Andrew M.; Carlier, Pierre G.; Spuler, Simone; Day, John W.; Jones, Kristi J.; Bharucha-Goebel, Diana X.; Salort-Campana, Emmanuelle; Pestronk, Alan; Walter, Maggie C.; Paradas, Carmen; Stojkovic, Tanya; Mori-Yoshimura, Madoka; Bravver, Elena; Diaz-Manera, Jordi; Pegoraro, Elena; Mendell, Jerry R.; Mayhew, Anna G. und Straub, Volker
(2021):
Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale.
In: Annals of Neurology, Bd. 89, Nr. 5: S. 967-978