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X-linked traits

Revised North Star Ambulatory Assessment for Young Boys with Duchenne Muscular Dystrophy

Eugenio Mercuri, Giorgia Coratti, [ ... ], Marika Pane

Apoptotic Activity of MeCP2 Is Enhanced by C-Terminal Truncating Mutations

Alison A. Williams, Vera J. Mehler, [ ... ], Carsten Duch

A Movement Monitor Based on Magneto-Inertial Sensors for Non-Ambulant Patients with Duchenne Muscular Dystrophy: A Pilot Study in Controlled Environment

Anne-Gaëlle Le Moing, Andreea Mihaela Seferian, [ ... ], Laurent Servais

Effect of the IL-1 Receptor Antagonist Kineret^® on Disease Phenotype in mdx Mice

Margaret E. Benny Klimek, Arpana Sali, [ ... ], Kanneboyina Nagaraju

C26:0-Carnitine Is a New Biomarker for X-Linked Adrenoleukodystrophy in Mice and Man

Malu-Clair van de Beek, Inge M. E. Dijkstra, [ ... ], Stephan Kemp

Natural History of Cardiac and Respiratory Involvement, Prognosis and Predictive Factors for Long-Term Survival in Adult Patients with Limb Girdle Muscular Dystrophies Type 2C and 2D

Abdallah Fayssoil, Adam Ogna, [ ... ], Djillali Annane

Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study

Simina M. Boca, Maki Nishida, [ ... ], Subha Madhavan

Rett Syndrome Mutant Neural Cells Lacks MeCP2 Immunoreactive Bands

Carlos Bueno, Rafael Tabares-Seisdedos, Jose M. Moraleda, Salvador Martinez

Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy

Valeria Ricotti, Stefan Spinty, [ ... ], Jonathon Tinsley

Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs

Mary Beth Callan, Mark E. Haskins, [ ... ], Valder R. Arruda

Timed Rise from Floor as a Predictor of Disease Progression in Duchenne Muscular Dystrophy: An Observational Study

Elena S. Mazzone, Giorgia Coratti, [ ... ], Eugenio Mercuri

Correlation of Utrophin Levels with the Dystrophin Protein Complex and Muscle Fibre Regeneration in Duchenne and Becker Muscular Dystrophy Muscle Biopsies

Narinder Janghra, Jennifer E. Morgan, [ ... ], Jonathon Tinsley

The mdx Mutation in the 129/Sv Background Results in a Milder Phenotype: Transcriptome Comparative Analysis Searching for the Protective Factors

Priscila Clara Calyjur, Camila de Freitas Almeida, [ ... ], Mariz Vainzof

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