Roche is developing an investigational medicine for SMA, called risdiplam.
The investigational treatment “risdiplam” (also known as RO7034067) is taken once daily in liquid form by mouth or feeding tube.
Risdiplam is called ‘investigational’ as it is not approved by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA). In our clinical trials we aim to learn whether risdiplam is safe and effective in people with Spinal Muscular Atrophy.
Risdiplam is developed in collaboration with the SMA Foundation and PTC Therapeutics.
FIREFISH clinical trial.
This is a clinical trial in babies aged between 1 to 7 months. The trial is ongoing, but is no longer open to new participants.
Find out more about FIREFISH here.
SUNFISH clinical trial.
The SUNFISH trial examines risdiplam in people aged 2 – 25 years with Type 2/3 SMA. The trial is ongoing, but is no longer open to new participants.
Find out more about SUNFISH here.
JEWELFISH clinical trial.
The JEWELFISH trial examines risdiplam in people between 6 months – 60 years of age with any Type of SMA. The clinical trial is for people who have previously participated in a trial with SMN2 targeting therapies or olesoxime or who received previously treatment with nusinersen.
Find out more about JEWELFISH here.