Novartis Gene Therapies
AveXis is now Novartis Gene Therapies. Novartis Gene Therapies is dedicated to developing and commercializing gene therapies for patients and families devastated by rare and life-threatening neurological genetic diseases.
Understanding Spinal Muscular Atrophy (SMA)
SMA is a rare and devastating genetic disease caused by a lack of a functional survival motor neuron 1 (SMN1) gene, resulting in the rapid and irreversible loss of motor neurons.
Newborn screening for SMA
In its most severe forms, spinal muscular atrophy can progress rapidly. Early diagnosis through newborn screening can help detect the disease before symptoms appear to aid early treatment intervention.
Projects in Novartis Gene Therapies pipeline
Projects in our clinical pipeline
Managed Access Program
The Novartis Gene Therapies "Managed Access Program" makes investigational or unapproved treatments available to eligible patients with serious or life-threatening diseases.