Genentech takes aim at Biogen and Novartis, highlighting an early look at positive results for their oral SMA drug from PTC
After getting hit with a setback that forced the pharma giant Roche to abandon its lead drug for spinal muscular atrophy, company execs are back with a new drug in-licensed from PTC $PTCT that they believe has legs.
It’s an interim, updated look at a tiny data set for risdiplam (or RG7916), but researchers spotlighted evidence that the drug could help significant percentages of infants sit, kick and control their head movements — all elements that the disease destroys as it relentlessly pushes these children to an early death.
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