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Journal Articles

Natural history study and statistical modeling of disease progression in a preclinical model of myotubular myopathy

Open Access
In collection:
Neuromuscular Disease
Suzie Buono, Arnaud Monseur, Alexia Menuet, Anne Robé, Catherine Koch, Jocelyn Laporte, Leen Thielemans, Marion Depla, Belinda S. Cowling
Journal: Disease Models & Mechanisms
Dis Model Mech (2022) 15 (7): dmm049284.
https://doi.org/10.1242/dmm.049284
Published: 25 July 2022
... therapeutic Dnm2 reduction in a dose–response analysis. Centronuclear myopathy Myotubular myopathy Preclinical disease model Dynamin Antisense oligonucleotide Therapy Neuromuscular disorder Dynacure Be Est Projets d'Avenir du programme d'Investissement Avenir 3 coDyn101...
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Includes: Supplementary data