dm+d

Unassigned

New Medicines

Duchenne muscular dystrophy

Information

New molecular entity
Catabasis Pharmaceuticals
Catabasis Pharmaceuticals

Development and Regulatory status

Discontinued
Discontinued
Discontinued
Yes
Yes
Oct 20Catabasis is stopping activities related to development of edasalonexent including ongoing GalaxyDMD open-label extension trial following failure to meet primary and secondary outcomes in PIII PolarisDMD trial [12].

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

Evidence based evaluations