The SMA Drug Pipeline

There are a number of drugs on the horizon that may be of potential therapeutic benefit to people living with SMA.

Using this SMA drug pipeline, we are tracking the development of possible SMA therapies currently being tested in human clinical trials. We have split the diagram into the drugs’ mode of action, whether they target the underlying genetics of SMA or if they protect the muscles or the motor neurons. This diagram will be regularly updated to map the development and progress of each drug programme.

This diagram shows the progress of drugs currently in clinical trials for the treatment of SMA: