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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
Acsadi, Gyula; Crawford, Thomas O.; Mueller-Felber, Wolfgang; Shieh, Perry B.; Richardson, Randal; Natarajan, Niranjana; Castro, Diana; Ramirez-Schrempp, Daniela; Gambino, Giulia; Sun, Peng und Farwell, Wildon
(2021):
Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study.
In: Muscle & Nerve, Bd. 63, Nr. 5: S. 668-677
Oh, Shin J.; Shcherbakova, Natalya; Kostera-Pruszczyk, Anna; Alsharabati, Mohammad; Dimachkie, Mazen; Munoz Blanco, Jose; Brannagan, Thomas; Lavrnic, Dragana; Shieh, Perry B.; Vial, Christophe; Meisel, Andreas; Komoly, Samuel; Schoser, Benedikt; Sivakumar, Kumaraswamy und So, Yuen
(2016):
Amifampridine phosphate (Firdapse((R))) is effective and safe in a phase 3 clinical trial in LEMS.
In: Muscle & Nerve, Bd. 53, Nr. 5: S. 717-725