Tamoxifen may contribute to preserve cardiac function in Duchenne muscular dystrophy
- Published on 08/30/2024
- Reading time: 3 min.
Bettina C. Henzi 1 ,6 na1, Sebastiano A. G. Lava 2 ,3 ,4 ,5 na1, Dirk Fischer 1
1 Division of Neuropediatrics and Developmental Medicine, University Children’s Hospital Basel, University of Basel, Basel, Switzerland
2 Pediatric Cardiology Unit, Department of Pediatrics, Centre Hospitalier Universitaire Vaudois and University of Lausanne, Rue du Bugnon 46, Lausanne, 1011, Switzerland
3 Heart Failure and Transplantation, Department of Paediatric Cardiology, Great Ormond Street Hospital, London, UK
4 Clinical Pharmacology and Therapeutics Group, University College London, London, UK
5 Division of Clinical Pharmacology and Toxicology, Institute of Pharmacological Sciences of Southern Switzerland, Ente Ospedaliero Cantonale, Lugano, Switzerland
6 Division of Neuropediatrics, Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital, and University of Bern, Bern, Switzerland
Abstract
Duchenne muscular dystrophy is life-limiting. Cardiomyopathy, which mostly ensues in the second decade of life, is the main cause of death. Treatment options are still limited. The TAMDMD (NCT03354039) trial assessed motor function, muscle strength and structure, laboratory biomarkers, and safety in 79 ambulant boys with genetically confirmed Duchenne muscular dystrophy, 6.5-12 years of age, receiving either daily tamoxifen 20 mg or placebo for 48 weeks. In this...
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