Duchenne muscular dystrophy (DMD): investigational treatments

Evidence-based neurology checklist on duchenne muscular dystrophy (dmd): investigational treatments: CAP-1002 These are intracoronary allogeneic cardiosphere-derived cells Their infusion reduces the size of myocardial fibrosis scar They also improve inferior wall systolic thickening Ezutromid (SMT…

CAP-1002

  • These are intracoronary allogeneic cardiosphere-derived cells
  • Their infusion reduces the size of myocardial fibrosis scar
  • They also improve inferior wall systolic thickening

Ezutromid (SMT C1100)

Edasalonexent

AdipoRon

Other investigational treatments

References

  1. Taylor M, Jefferies J, Byrne B, et al. Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial. Neurology 2019; 92:e866-e878.
  2. Muntoni F, Tejura B, Spinty S, et al. A phase 1b trial to assess the pharmacokinetics of ezutromid in pediatric Duchenne muscular dystrophy patients on a balanced diet. Clin Pharmacol Drug Dev 2019; 8:922-933.
  3. Finanger E, Vandenborne K, Finkel RS, et al. Phase 1 study of edasalonexent (CAT-1004), an oral NF-κB inhibitor, in pediatric patients with Duchenne muscular dystrophy. J Neuromuscul Dis 2019; 6:43-54.
  4. Finkel RS, McDonald CM, Lee Sweeney H, et al. A Randomized, double-blind, placebo-controlled, global phase 3 study of edasalonexent in pediatric patients with Duchenne muscular dystrophy: results of the PolarisDMD Trial. J Neuromuscul Dis 2021 (Online ahead of print).
  5. Abou-Samra M, Selvais CM, Boursereau R, Lecompte S, Noel L, Brichard SM. AdipoRon, a new therapeutic prospect for Duchenne muscular dystrophy. J Cachexia Sarcopenia Muscle 2020; 11:518-533.
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