Roche is developing an investigational medicine for SMA, risdiplam.
Risdiplam (also known as RO7034067) is developed in collaboration with the SMA Foundation and PTC Therapeutics. Risdiplam is given by mouth or feeding tube.
In our FIREFISH, SUNFISH and JEWELFISH trials we aim to learn if risdiplam is safe and effective.
Our clinical trial for Type 1 SMA is called FIREFISH.
This is a clinical trial in babies aged between 1 to 7 months.
Find out more about FIREFISH here.
Our clinical trials for SMA Type 2 and Type 3 are called SUNFISH and JEWELFISH.
The SUNFISH trial examines risdiplam in people aged 2 – 25 years with Type 2/3 SMA.
Find out more about SUNFISH here.
The JEWELFISH trial examines risdiplam in people aged 12 – 60 years with Type 2/3 SMA. The clinical trial is for people who have previously participated in a trial with SMN2 targeting therapies.
Find out more about JEWELFISH here.