Safety and efficacy of delpacibart zotadirsen in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the USA (EXPLORE44): a randomised, placebo-controlled, double-blind, phase 1/2 trial.
Delpacibart zotadirsen shows potential safety and efficacy for treating specific Duchenne muscular dystrophy mutations in a randomized trial.
This PubMed record reports a randomized controlled trial about Safety and efficacy of delpacibart zotadirsen in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the USA (EXPLORE44): a randomised, placebo-controlled, double. The available abstract indicates Avidity Biosciences, A Novartis Company.
What the study was
- Study design
- Randomized controlled trial
- Population
- Human participants
- Category
- Treatment Innovation
- Maturity
- Potentially Practice-Changing
- Journal
- The Lancet. Neurology
Why it surfaced
Randomized controlled trial; directly matches Rare diseases with high unmet need. Conservative abstract-level score 9/10.
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