Novartis Gene Therapies is dedicated to developing and commercializing gene therapies for patients and families devastated by rare and life-threatening neurological genetic diseases.

About Novartis Gene Therapies

Since 2013, Novartis Gene Therapies (formerly AveXis) has had one focus: bringing change to those devastated by genetic diseases.

Gene therapy manufacturing

Novartis Gene Therapies has developed a reproducible manufacturing process to address rare genetic disorders.

Resources for the rare disorders patient community

Novartis Gene Therapies is proud to stand as an ally with the patient community.

20+

Projects in our gene therapy pipeline

162

Projects in our clinical pipeline

Novartis pipeline

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.

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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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Novartis Gene Therapies request

Novartis Gene Therapies wants to ensure that physicians, researchers, and other healthcare professionals have access to complete, up-to-date, and balanced scientific information regarding gene therapy treatment, research and investigational therapies.

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Contact Novartis Gene Therapies

Please direct any of your medical inquiries to the Novartis Gene Therapies Medical Information in the US (Healthcare Professionals, Consumers), Europe, Middle East and Africa, Latin America and Canada, or Asia-Pacific and Australia. Visit the Novartis Gene Therapies Medical Information website for additional information.

Side effects of Novartis medicines can be reported online or to local Novartis Drug Safety Responsible at novartis.com/report.

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.

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