Tamoxifen in children with Duchenne muscular dystrophy

Duchenne muscular dystrophy is a severe neuromuscular disorder that affects about one in 5000 male newborn babies. 1 Markati T Oskoui M Farrar MA et al. Emerging therapies for Duchenne muscular dystrophy. Lancet Neurol. 2022; 21: 814-819 Summary Full Text Full Text PDF PubMed Google Scholar In June, 2023, the US Food and Drug Administration conditionally approved gene therapy with adeno-associated virus microdystrophin for boys aged 4–5 years; the phase 3 trial is ongoing. 2 Mendell JR Sahenk Z Lehman K et al. Assessment of systemic delivery of rAAVrh74.MHCK7.micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial. JAMA Neurol. 2020; 77: 1122-1131 Crossref PubMed Scopus (163) Google Scholar Despite this major milestone, treatments for boys with Duchenne muscular dystrophy are urgently needed. Furthermore, the magnitude and duration of effect of gene therapy, and the optimal age at which to deliver it, are uncertain. Moreover, with a current price of about US$3 200 000, this genetic treatment is unlikely to be equally available worldwide. Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trialTamoxifen was safe and well tolerated, but no difference between groups was reported for the primary efficacy endpoint. Slower disease progression, defined by loss of motor function over time, was indicated in the tamoxifen group compared with the placebo group, but differences in outcome measures were neither clinically nor statistically significant. Currently, we cannot recommend the use of tamoxifen in daily clinical practice as a treatment option for boys with Duchenne muscular dystrophy due to insufficient clinical evidence. Full-Text PDF