A recording of this week’s webinar with Genentech/Roche is now available online.
During the webinar, Genentech/Roche shared their history working with the SMA community, and their investigational medicine risdiplam. Updates on their clinical trials in pre-symptomatic & types 1, 2 & 3 were also shared.
Risdiplam is an investigational, oral medicine that is systemically distributed and designed to increase SMN protein levels in the central nervous system (CNS) and throughout the body. It is designed to help the SMN2 gene produce more functional SMN protein, to better support motor neurons and muscle function. Roche and Genentech are leading the clinical development of risdiplam in collaboration with the SMA Foundation and PTC Therapeutics.