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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About Novartis Gene Therapies

Since2013，Novartis Gene Therapies（formerly AveXis）has had one focus:bringing change to those devastated by genetic diseases。
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Gene therapy manufacturing

Novartis Gene Therapies has developed areproducible manufacturing process to address rare genetic disorders。
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按需提供资源

Novartis Gene Therapies is proud to stand as an ally with the patient community。
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Novartis pipeline
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Spinal Muscular Atrophy（SMA）

SMA is a rare and devastating genetic disease caused by a lack of a functional survival motor neuron1（SMN1）gene，resulting in the rapid and irreversible loss of motor neurons。

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SMA为Newborn screening

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单击功能区上，消费者，消费者）Europe，Middle East and Africa单击功能区上Latin America and Canada，orAsia-Pacific and Australia.Visit theNovartis Gene Therapies Medical Information websitefor additional information。

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管理的访问程序

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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