Press Release: New Novartis Phase III data -2-


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About Novartis

Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide.

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References

1. Weber C, et al. 'Reading the palm' -- A pilot study of grip and finger

flexion strength as an outcome measure in 5q spinal muscular atrophy.

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Brain and Development. 2024;46(5):89-198

2. Coratti G et al. Determining minimal clinically important differences in

the Hammersmith Functional Motor Scale Expanded for untreated spinal

muscular atrophy patients: An international study. Eur J Neurol.

2024;31:e16309.

3. Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific

clinical outcome assessment tool - PMC.

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4. O'Hagen JM, Glanzman AM, McDermott MP, Ryan PA, Flickinger J, Quigley J,

et al. An expanded version of the Hammersmith Functional Motor Scale for

SMA II and III patients. Neuromuscular disorders: NMD.

2007;17(9--10):693--7. Epub 2007/07/31.

5. Hammersmith Functional Motor Scale and Motor Function Measure-20 in non

ambulant SMA patients - PubMed. Mazzone E, De Sanctis R, Fanelli L,

Bianco F, Main M, van den Hauwe M, et al. Hammersmith Functional Motor

Scale and Motor Function Measure-20 in non ambulant SMA patients.

Neuromuscular disorders: NMD. 2014;24(4):347--52. Epub 2014/02/05.

10.1016/j.nmd.2014.01.003.

6. Basil T. Darras. Intravenous and Intrathecal Onasemnogene Abeparvovec

Gene Therapy in Symptomatic and Presymptomatic Spinal Muscular Atrophy:

Long-Term Follow-Up Study. Presented at the 29th International Annual

Congress of the World Muscle Society, October 8--12, 2024, Prague,

Czechia.

7. Clinicaltrials.Gov. Available at:

https://clinicaltrials.gov/study/NCT05089656. Accessed February 2025.

8. Finkel RS, et al. J Neuromuscul Dis. 2023;10(3):389-404.

9. Clinicaltrials.Gov. Available at:

https://clinicaltrials.gov/study/NCT03381729. Accessed November 2024.

10. Clinicaltrials.Gov. https://clinicaltrials.gov/study/NCT05386680.

Accessed November 2024.

11. Anderton RS and Mastaglia FL. Expert Rev Neurother. 2015;15(8):895--908.

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

13. Lorson CL, et al. Hum Mol Genet. 2010;(15):111-8.

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(END) Dow Jones Newswires

March 19, 2025 09:00 ET (13:00 GMT)

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