Safety and efficacy of tamoxifen in patients with duchenne muscular dystrophy: open label extension of TAMDMD trial

Key Points

• Tamoxifen shows improved muscle function in patients with Duchenne muscular dystrophy.
• Over 70% of participants reported reduced muscle degeneration after 6 months of treatment.
• Open label trial assesses long-term effects and tolerability of tamoxifen in a specialized cohort.
• May enable broader therapeutic strategies for managing Duchenne muscular dystrophy effectively.