Novartis, shadowed by data scandal, renews case for Zolgensma

Infants likely to develop a severe form of spinal muscular atrophy, but who were not yet symptomatic, could sit and stand following treatment with Novartis' gene therapy Zolgensma, according to new study results disclosed by the Swiss drugmaker. The data, presented Thursday at the European Paediatric Neurology Society's annual meeting in Athens, Greece, are an update from an initial glimpse the company gave in May. Since then, more babies with the inherited neuromuscular disease achieved motor milestones like sitting independently for more than 30 seconds or standing with assistance. Typically, infants diagnosed with severe SMA are never able to sit, and most die by age two. Five patients in the trial, called SPR1NT, achieved the maximum score on a University of Pennsylvania scale used to gauge motor abilities, an improvement from the three who did by the May update. Two others have reached normal levels.