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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
Schoser, Benedikt; Bilder, Deborah A.; Dimmock, David; Gupta, Digant; James, Emma S. und Prasad, Suyash
(2017):
The humanistic burden of Pompe disease: are there still unmet needs? A systematic review.
In: BMC Neurology
17:202
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