Risdiplam Clinical Trials and Their Results
Risdiplam Clinical Trials and Their Results
Page last checked: 21st December 2022
Last updated: 18th February 2022
The first in-human trials of risdiplam, which were conducted in healthy volunteers (i.e. a Phase 1 clinical trial), were initiated in late 2015, with the first volunteers enrolled in early 2016.
SMA patients were first enrolled in clinical trials of risdiplam in late 2016: the FIREFISH trial involving infants with SMA Type 1, and the SUNFISH trial involving participants with SMA Type 2 and 3. JEWELFISH and RAINBOWFISH trials followed later.
Decisions by regulatory authorities to agree access to risdiplam have drawn on the results of these four clinical trials.
Results and Updates for Each Trial >
Clinical Trial Timeline for all Risdiplam Trials >
In April 2023, Our Scientific Correspondent collated the following: