Tamoxifen in children with Duchenne muscular dystrophy

Journal article : Comment

Duchenne muscular dystrophy is a severe neuromuscular disorder that affects about one in 5000 male newborn babies. 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. 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.

Authors: Servais, L

• Publication status: Published
• Peer review status: Reviewed (other)
• Publisher: Elsevier
• Journal: The Lancet Neurology
• Volume: 22
• Issue: 10
• Pages: 872-873
• Publication date: 2023-10-01
• Acceptance date: 2023-07-24
• DOI: 10.1016/s1474-4422(23)00288-0
• EISSN: 1474-4465
• ISSN: 1474-4422
• Pmid: 37739562
• Language: English
• Subtype: Comment