24.05.2019 18:30:53

Press Release: Novartis: AveXis receives FDA -3-

bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. In

addition to developing Zolgensma to treat all forms of SMA, AveXis also

plans to develop other novel treatments for rare neurological diseases,

including Rett syndrome and a genetic form of amyotrophic lateral

sclerosis caused by mutations in the superoxide dismutase 1 (SOD1) gene.

For additional information, please visit www.avexis.com.

About Novartis

Novartis is reimagining medicine to improve and extend people's lives.

As a leading global medicines company, we use innovative science and

digital technologies to create transformative treatments in areas of

great medical need. In our quest to find new medicines, we consistently

rank among the world's top companies investing in research and

development. Novartis products reach more than 750 million people

globally and we are finding innovative ways to expand access to our

latest treatments. About 105 000 people of more than 140 nationalities

work at Novartis around the world. Find out more at www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at

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For Novartis multimedia content, please visit

www.novartis.com/news/media-library

For questions about the site or required registration, please contact

media.relations@novartis.com

References

[1] Anderton RS and Mastaglia FL. Expert Rev Neurother.

2015;15(8):895-908.

[2] National Organization for Rare Disorders (NORD). Spinal Muscular

Atrophy. http://rarediseases.org/rarediseases/spinal-muscular-atrophy/.

Accessed October 9, 2018.

[3] Finkel RS, et al. Neurology. 2014;83(9):810-7.

[4] Farrar MA, et al. Ann Neurol. 2017;81(3):355-368.

[5] Data on file.

[6] Verhaart IEC, Robertson A, et al. J Neurol. 2017

Jul;264(7):1465-1473.

[7] Soler-Botija C, et al. Brain. 2002;125(7):1624-1634.

[8] Swoboda KJ, et al. Ann Neurol. 2005; 57(5):704-712.

[9] Administration HRaS. Recommended Uniform Screening Panel (RUSP).

2018;

https://www.hrsa.gov/advisory-committees/heritable-disorders/rusp/index.html.

Accessed October 30, 2018, 2018.

# # #

Novartis Media Relations

Central media line: +41 61 324 2200

E-mail: media.relations@novartis.com

Eric Althoff Farah Bulsara Speer

Novartis External Communications VP, Corporate Communications, AveXis

+1 646 438 4335 (mobile) +1 312 543 2881 (mobile)

eric.althoff@novartis.com fSpeer259@avexis.com

Novartis Investor Relations

Central investor relations line: +41 61 324 7944

E-mail: investor.relations@novartis.com

Central North America

Samir Shah +41 61 324 7944 Richard Pulik +1 862 778 3275

Pierre-Michel Bringer +41 61 324 1065 Cory Twining +1 862 778 3258

Thomas Hungerbuehler +41 61 324 8425

Isabella Zinck +41 61 324 7188

Media release (PDF): http://hugin.info/134323/R/2244903/886818.pdf

This announcement is distributed by West Corporation on behalf of West

Corporation clients.

The issuer of this announcement warrants that they are solely

responsible for the content, accuracy and originality of the information

contained therein.

Source: Novartis International AG via Globenewswire

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Novartis International AG

P.O. Box Basel Switzerland

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http://www.novartis.com

(END) Dow Jones Newswires

May 24, 2019 12:31 ET (16:31 GMT)

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