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.

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

A lay explanation of the results obtained through Phase 1 clinical trials can be found below:

Resources

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

Community Updates:

Clinical trials registers: