Roche’s risdiplam

Roche’s small molecule, risdiplam (RG7916), is an orally-available compound developed in collaboration with the SMA Foundation and PTC Therapeutics and which is used to increase the amount of SMN protein made by the SMN2 gene.

A lay explanation of what risdiplam is and the results obtained through Phase 1 clinical trials can be found here.

There are currently 4  trials for risdiplam, which started in December 2016 in SMA Types 1, 2 & 3:

 Study NameSMA PopulationAge
FIREFISHType 1Between 1 & 7 months
SUNFISHType 2 & 3Between 2 & 25 years
JEWELFISHType 1, 2 & 3Between 6 months & 60 years and has received nusinersen or participated in trial of other SMN-targeting therapy or olesoxime
RAINBOWFISHPresymptomaticUp to 6 weeks



Roche has a dedicated website for these SMA trials, where more information can be found.

Community Updates:

Clinical trials registers: