Novartis Gene Therapies
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.
Projects in our gene therapy pipeline
Projects in our clinical 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.
Newborn screening for SMA
<p>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.</p>
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.
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 theNovartis Gene Therapies Medical Information website for additional information.
Side effects of Novartis medicines can be reported online or to local Novartis Drug Safety Responsible atnovartis.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.