Meeting Report : 2022 Muscular Dystrophy Association Summit on 'Safety and Challenges in Gene Transfer Therapy'

enthalten in: Journal of neuromuscular diseases | 2023

von Lek, A. | Atas, E. | Hesterlee, S. | +2

Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy : A qualitative preference study of patients and parents

enthalten in: PloS one | 2019

von Landrum Peay, H. | Fischer, R. | Tzeng, J. | +9

A call for transparent reporting to optimize the predictive value of preclinical research

enthalten in: Nature | 2012

von Landis, S. | Amara, S. | Asadullah, K. | +33

Whole-genome analysis of sporadic amyotrophic lateral sclerosis

enthalten in: The New England journal of medicine | 2007

von Dunckley, T. | Huentelman, M. | Craig, D. | +32

Challenges in drug development for muscle disease : a stakeholders' meeting

enthalten in: Muscle & nerve | 2007

von Mendell, J. | Csimma, C. | McDonald, C. | +5

Challenges in drug development for muscle disease: A stakeholders' meetingFNR HREF="fn1">FN ID="fn1">Report of a meeting sponsored by the Muscular Dystrophy Association, August 4-5, 2005 Bethesda, Maryland, USA.

enthalten in: Muscle & nerve | 2007

von Mendell, J. | Csimma, C. | Mcdonald, C. | +5