##  [Novartis Gene Therapies](/about/novartis-gene-therapies "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.

 

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 ![Scientists Viewing Monitors](/sites/novartis_com/files/styles/cards_1_3/public/2021-10/scientists-viewing-monitors.jpg.webp?itok=j6zFERWW "Scientists Viewing Monitors") 

 

###  About Novartis Gene Therapies 

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

 

 

 ](/about/novartis-gene-therapies/about-novartis-gene-therapies) [ 

 ![scientist pipetting](/sites/novartis_com/files/styles/cards_1_3/public/2021-10/scientist-pipetting.jpg.webp?itok=IWi83xGm "scientist pipetting") 

 

###  Gene therapy manufacturing 

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

 

 

 ](/about/novartis-gene-therapies/gene-therapy-manufacturing) [ 

 ![Patient using a tablet](/sites/novartis_com/files/styles/cards_1_3/public/2021-09/patient-image-using-a-tablet.jpg.webp?itok=YiHtkTMO "Patient using a tablet") 

 

###  Resources for the rare disorders patient community 

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

 

 

 ](/about/novartis-gene-therapies/resources-rare-disorders-patient-community) 





 

 

 



 20+ 

####  Projects in our gene therapy pipeline 

 

 162 

####  Projects in our clinical pipeline 

 

 

 [Novartis pipeline](/research-development/novartis-pipeline "Novartis pipeline") 





    ![Parents snuggling with their son](/sites/novartis_com/files/styles/cosmos_banner_half_image_large/public/2021-10/parents-snuggling-with-son-sma.jpg.webp?itok=WjWRu7VX "Parents snuggling with their son") 

 

 

 

##  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.

 [Learn more](/diseases/spinal-muscular-atrophy-sma "Learn more") 

 

 

 



 

 

 

 



 ![Mum holding newborn's feet](/sites/novartis_com/files/styles/cta_image/public/2021-10/mum-holding-newborns-feet.jpg.webp?itok=MJGJXvWI "Mum holding newborn's feet") 

 

##  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>



 

 [Learn more](/diseases/spinal-muscular-atrophy-sma/newborn-screening-spinal-muscular-atrophy-sma "Learn more") 



 

 



 





### 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.

[Learn more](/about/novartis-gene-therapies/novartis-gene-therapies-request-funding "Novartis Gene Therapies Request for Funding")



### Contact Novartis

Please direct any of your medical inquiries to the Novartis Medical Information in the US ([Healthcare Professionals](/healthcare-professionals/novartis-medical-information "Novartis medical information"), [Consumers](https://www.novartis.com/us-en/about/contact-us)), [Europe, Middle East and Africa](/healthcare-professionals/novartis-medical-information "Novartis medical information"), [Latin America and Canada](/healthcare-professionals/novartis-medical-information "Novartis medical information"), or [Asia-Pacific and Australia](/healthcare-professionals/novartis-medical-information "Novartis medical information").

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

[Learn more](/healthcare-professionals/managed-access-programs/zolgensma-global-managed-access-program-gmap "Zolgensma® Global Managed Access Program (gMAP)")