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Eine Ebene höherGruppiert nach: Dokumententyp |
Veröffentlichungsdatum
Anzahl der Publikationen: 5
2022
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
2021
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
2019
Schoser, Benedikt; Hahn, Andreas; James, Emma; Gupta, Digant; Gitlin, Matthew und Prasad, Suyash
(2019):
A Systematic Review of the Health Economics of Pompe Disease.
In: Pharmacoeconomics-Open, Bd. 3, Nr. 4: S. 479-493
2017
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
[PDF, 873kB]
2016
Lawlor, Michael W.; Beggs, Alan H.; Buj-Bello, Ana; Childers, Martin K.; Dowling, James J.; James, Emma S.; Meng, Hui; Moore, Steven A.; Prasad, Suyash; Schoser, Benedikt und Sewry, Caroline A.
(2016):
Skeletal Muscle Pathology in X-Linked Myotubular Myopathy: Review With Cross-Species Comparisons.
In: Journal of Neuropathology and Experimental Neurology, Bd. 75, Nr. 2: S. 102-110