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:
Between 1 & 7 months
Type 2 & 3
Between 2 & 25 years
Type 1, 2 & 3
Between 6 months & 60 years and has received nusinersen or participated in trial of other SMN-targeting therapy or olesoxime