Casimersen

Unassigned

New Medicines

Duchenne muscular dystrophy with 45‐exon skipping activity in patients aged 7 to 16 years

Information

New molecular entity
Sarepta Therapeutics
Sarepta Therapeutics

Development and Regulatory status

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

Category

Weekly administered RNA‐based therapeutic and phosphorodiamidate morpholino oligomers (PMOs) which bind to RNA and efficiently interfere with gene expression in a sequence-specific manner.
DMD affects approximately 1 in every 3,500 boys that are born but only around 1 in 50 million girls [1]. In the UK, about 100 boys are born with DMD each year, and there are about 2,500 boys living with the condition at any one time [2].
Duchenne muscular dystrophy with 45‐exon skipping activity in patients aged 7 to 16 years
Intravenous infusion