Related Experiment Video
Updated: Aug 2, 2025
09:18
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
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Utrophin correlates with disease severity in Duchenne muscular dystrophy
1Paris-Saclay University, UVSQ, INSERM-UMR1179, END-ICAP Laboratory, 78000 Versailles, France; SQY Therapeutics, 78180 Montigny-Le-Bretonneux, France.
Med (New York, N.Y.)
|April 15, 2023
Abstract:
This month in Med, the description of an unusually severely affected DMD patient suffering from a large deletion in the dystrophin gene confirms that absence of utrophin worsens the dystrophy and supports the concept that utrophin upregulation ameliorates the pathology. This study may guide the development of dystrophin-based gene therapies.
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