Roche (OTCQX:RHHBY) announces positive results from Part 2 of a Phase 2/3 clinical trial, FIREFISH, evaluating risdiplam in infants ages 1-7 months with spinal muscular atrophy type 1 (SMA1).
The study met the primary endpoint of the
proportion of infants sitting without support for at least five seconds
at month 12 as measured by a scale called BSID-III.
No new safety signals were observed.
Detailed data will be submitted for presentation at a future medical conference.
The company announced positive results from another Phase 2/3 study, SUNFISH, in November 2019.
Risdiplam is a survival motor neuron-2 (SMN2)
splicing modifier designed to increase and sustain SMN protein levels
throughout the central nervous system and in peripheral tissues.
Roche is leading development in collaboration with PTC Therapeutics (NASDAQ:PTCT) and the SMA Foundation.
https://seekingalpha.com/news/3533806-roches-risdiplam-successful-in-sma-study
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.