Nearly four years on since its US approval, Novartis’s gene therapy Zolgensma has transformed and extended lives of several thousand infants born with the most severe form of spinal muscular atrophy (SMA) who would otherwise have died before their second birthday.
Novartis’s ‘Poster Child’ Zolgensma Shows What Gene Therapy Can Do – And Its Limitations
Long-Term Data Brings Hope To Rare Disease Communities
The clinical development head of Novartis Gene Therapies talks about the progress for SMA patients more than seven years after treatment began, and hopes of boosting outcomes by treating pre-symptomatic patients.
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