Novartis Gene Therapies
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.
AveXis renamed Novartis Gene Therapies, signifying the growing importance of gene therapy to Novartis corporate strategy
About Novartis Gene Therapies
Since 2013, Novartis Gene Therapies (formerly AveXis) has had one focus: bringing change to those devastated by 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.
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6
Projects in Novartis Gene Therapies pipeline
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162
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.
Gene Therapy Manufacturing
Novartis Gene Therapies has developed a reproducible manufacturing process to address rare genetic disorders.
Please direct any of your medical inquiries to the Novartis Gene Therapies MedInfo team in the US, Europe, Middle East and Asia, Latin America & Canada, or Asia-Pacific.
Side effects of Novartis medicines can be reported online or to local Novartis Drug Safety Responsible at report.novartis.com.