Drug pipeline • World Duchenne (original) (raw)

Here, an overview of potential compounds tested for the treatment of Duchenne muscular dystrophy is given. This includes compounds aiming to restore/ replace the missing dystrophin and compounds targeting one or more of the secondary effects (e.g. fibrosis of inflammation) of the absence of dystrophin.

Conditional approval

If a drug gained conditional approval, this is marked with an asterisk (*). Conditional approval means that the drug is approved, but that this is only valid for a certain period, usually one year. After this, the company has to meet certain conditions (e.g. conduct additional trials, show new data) to renew the (conditional) approval.