When Yuliya Matyushenko was 12 years old, the Paris-based rare disease organization Eurordis featured her on a poster promoting Rare Disease Day 2015. In late February, the young woman with spinal muscular atrophy (SMA) was hiding in a bare concrete basement in Kharkiv as Ukraine’s second-largest city, located only 35 kilometers from the Russian border, . . .
On Aug. 7, the US Food and Drug Administration (FDA) granted approval of risdiplam (Evrysdi) for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. It is the third disease-modifying therapy approved to treat SMA, the leading genetic cause of infant death. The drug is an orally administered liquid, the first medicine that could . . .
On Jan. 22, Genentech, a member of the Roche Group, announced positive results from the second part of its pivotal phase 2/3 FIREFISH clinical trial assessing risdiplam in infants age 1 to 7 months with spinal muscular atrophy (SMA) type 1. The company announced that the study met its primary endpoint, which was a proportion of infants . . .
On Nov. 25, Genentech, a member of the Roche Group, announced that the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for risdiplam, an investigational therapy for the treatment of spinal muscular atrophy (SMA). Priority Review status requires the FDA to review the application and decide on whether . . .
On Nov. 10, Genentech, a member of the Roche Group, announced positive results from the second part of its pivotal phase 2/3 SUNFISH clinical trial assessing risdiplam in patients with spinal muscular atrophy (SMA) types 2 or 3. The study met its primary endpoint, which was change from baseline in the Motor Function Measure 32 (MFM-32) . . .
Several biotech and pharma companies presented interim results at the 24th Annual Congress of the World Muscle Society (WMS) held earlier this month in Copenhagen, Denmark. This roundup covers a few of these updates. Catabasis Pharmaceuticals presented positive results from its phase 1/2 MoveDMD trial and open-label extension with edasalonexent in boys with Duchenne muscular . . .
