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Safety and efficacy of tamoxifen in patients with duchenne muscular dystrophy: open label extension of TAMDMD trial | Synapse
March 3, 2026
Safety and efficacy of tamoxifen in patients with duchenne muscular dystrophy: open label extension of TAMDMD trial
GZ
Gaëtan Zwingli
NP
Niveditha Putananickal
SS
Simone Schmidt
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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.
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Zwingli et al. (Sun,) studied this question.
synapsesocial.com/papers/69a7657cbadf0bb9e87d94c2
https://doi.org/https://doi.org/10.1016/j.nmd.2026.106366