Edasalonexent

Unassigned

New Medicines

Duchenne muscular dystrophy

Information

New molecular entity
Catabasis Pharmaceuticals
Catabasis Pharmaceuticals

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

NF-kappa B inhibitor, modulating transcription of pro- and anti-inflammatory mediators
The incidence of DMD is approximately 1 in 3,600-6,000 male births per year. In the UK, there are approximately 100 boys diagnosed with DMD each year & at any one time there are about 1,500 known to have the disease. Approximately 10-15% of patients have a nonsense mutation, which equates to between 150 and 195 patients in the UK.
Duchenne muscular dystrophy
Oral