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Dorchies, O., Neff, L., Gayi, E., Baulet, C., Fabien, S., de Mestral, R., ... & Scapozza, L. (2019). DMD CLINICAL AND BIOMARKERS: P. 139Paediatric use of tamoxifen: good safety profile for boys with Duchenne muscular dystrophy and X-linked myotubular myopathy. Neuromuscular Disorders, 29, S88-S89.
Dorchies, O., et al. "DMD CLINICAL AND BIOMARKERS: P. 139Paediatric use of tamoxifen: good safety profile for boys with Duchenne muscular dystrophy and X-linked myotubular myopathy." Neuromuscular Disorders 29 (2019): S88-S89.
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O Dorchies, L Neff, E Gayi, C Baulet… - Neuromuscular …, 2019 - nmd-journal.com

DMD CLINICAL AND BIOMARKERS: P. 139Paediatric use of tamoxifen: good safety profile for boys with Duchenne muscular dystrophy and X-linked myotubular ….

STEM and boys

Gayi

GSID: ByphKFzWGN4J

Excerpt

Duchenne muscular dystrophy (DMD) is caused by the lack of dystrophin, a protein that connects the extracellular matrix to the cytoskeleton in muscle fibres. DMD boys develop progressive muscle wasting, leading to cardiac and respiratory failure and premature death. X-linked myotubular (centronuclear) myopathy (XLMTM) is a very severe myopathy characterized by generalized hypotonia from birth and death of most patients before the age of 2. Our recent preclinical data in mice demonstrated that tamoxifen (a drug used against …

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