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Molecular Therapy. Methods & Clinical Development|July 23, 2020
Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular DystrophyVirginie Mariot, Caroline Le Guiner, Inès Barthélémy, et al.
Neuromuscular Disorders : NMD|May 13, 2014
Proteomics profiling of urine reveals specific titin fragments as biomarkers of Duchenne muscular dystrophyJeremy Rouillon, Aleksandar Zocevic, Thibaut Leger, et al.
BMC Musculoskeletal Disorders|April 15, 2011
Longitudinal ambulatory measurements of gait abnormality in dystrophin-deficient dogsInès Barthélémy, Eric Barrey, Pablo Aguilar, et al.
Archives of Neurology|February 16, 2006
Spectrum of brain changes in patients with congenital muscular dystrophy and FKRP gene mutationsEugenio Mercuri, Haluk Topaloglu, Martin Brockington, et al.
Neurogenetics|November 2, 2004
Early onset autosomal dominant spastic paraplegia caused by novel mutations in SPG3AAnnette Abel, Nuria Fonknechten, Anne Hofer, et al.
Skeletal Muscle|December 10, 2016
Skeletal muscle characteristics are preserved in hTERT/cdk4 human myogenic cell linesMatthew Thorley, Stéphanie Duguez, Emilia Maria Cristina Mazza, et al.
Molecular Therapy. Nucleic Acids|March 4, 2018
RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target EffectClaire Domenger, Marine Allais, Virginie François, et al.
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