Roche (OTCQX:RHHBY) unit Genentech announces positive results from two clinical trials evaluating risdiplam in patients with spinal muscular atrophy (SMA).
An exploratory analysis of the Phase 2/3 SUNFISH study in patients with type 2 or 3 SMA showed that risdiplam significantly improved motor function after 24 months of treatment.
Preliminary 12-month data from the Phase 2 JEWELFISH trial
in patients with all SMA types showed that treatment with risdiplam led
to rapid and sustained increases in survival motor neuron (SMN) protein
levels.
No new safety signals were observed.
The company’s U.S. marketing application is currently under FDA review with an action date of August 24.
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.
Related ticker: PTC Therapeutics (NASDAQ:PTCT)
https://seekingalpha.com/news/3582789-roches-risdiplam-shows-treatment-benefit-in-sma-studies
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