Duchenne muscular dystrophy (DMD): investigational treatments

Written and reviewed by Dr Ibrahim Imam, Consultant Neurologist. Last reviewed .

Duchenne muscular dystrophy (DMD): investigational treatments. These are intracoronary allogeneic cardiosphere-derived cells.

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)

Locked: subscribe to see this section.

Edasalonexent

Locked: subscribe to see this section.

AdipoRon

Locked: subscribe to see this section.

Other investigational treatments

Locked: subscribe to see this section.

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.
  6. And 4 more. Subscribe to see the full list

Related checklists

Loading...