Spinraza was the beginning. Roche is in the development of a small molecule named RG7916 a new therapy for SMA. ‘Hope with Roche #SMA #SpinalMuskelAtrofi
▶ Clinical Trials with RG7916 by Roche
FIREFISH TRIAL FOR SMA TYPE 1
A NEW TOOL FOR TRACKING THE PROGRESSION OF SPINAL MUSCULAR ATROPHY (SMA): QUANTITATIVE MAGNETIC RESONANCE IMAGING (QMRI).
Community Update 08-08-2017
Community Update 15-08-2017
▶ Community Update 15-3-2018
SMN protein increased by up to two and a half times. This increase has been sustained throughout the duration of treatment (up to 250 days). To date, RG7916 remains well-tolerated at all doses and no-one has withdrawn from any RG7916 study due to drug related side effects. The information from Part 1 has allowed us to confirm the dose of RG7916 for Part 2 of SUNFISH and the first patient has been enrolled into Part 2.
Firefish Efficacy of RO7034067 in Infants With SMA Type1
Jewelfish A Study of RO7034067
Sunfish A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in SMA Type 2 and 3
First Publication 29. Aug 2016 UPDATED Nils