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Oct 3, 2026

Z-basivarsen shows sustained benefits in myotonic dystrophy type 1 trial

An investigational drug, z-basivarsen, demonstrated sustained improvements in myotonia, motor function, and strength in patients with myotonic dystrophy type 1, according to analysis from the ACHIEVE trial. The findings suggest potential therapeutic benefits for this genetic neuromuscular disorder.

Oct 3, 2026·2 sources
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Top claims

  • ▪The FDA should grant accelerated approval to z-basivarsen for myotonic dystrophy
  • ▪Treatment-related adverse events for z-basivarsen in the ACHIEVE trial included infusion-related reactions, nasopharyngitis, diarrhea, headache, procedural pain, influenza, and back pain.
  • ▪Video hand-opening time is an adequate surrogate endpoint for regulatory approval of myotonic dystrophy drugs

Subtopics

Clinical trials1Drug development1Rare diseases1

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