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FULLSCREEN > Archive > 2019 > №1 (2019) > Spinal muscular atrophy: unlocking the potential of patients

Spinal muscular atrophy: unlocking the potential of patients

Various authors

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  • Abstract
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Abstract

In June, the International Congress on Pediatric Neurology was held. As part of an extensive program, a symposium "Spinraza: Unlocking the potential of patients," was held, during which 3 reports were presented on the management of patients with spinal muscular atrophy.

Key words: spinal muscular atrophy, therapy, spinraza.


About the Author

Various authors

References

1. SJ. Kolb, JT. Kissel Spinal Muscular Atrophy. A Timely Review. Arch Neurol. 2011 Aug; 68(8)

2. J Montes et al. Clinical outcome measures in Spinal Muscular atrophy. Journal of Child Neurology

3. Lunn MR. Spinal muscular atrophy. Lancet. 2008;371:2120-33

4. Tizzano EF and Finkel RS. Spinal muscular atrophy: A changing phenotype beyond the clinical trials. Neuromusc Disord. 2017;27:883–889.

5. Finkel R.S., Mercuri E., Meyer O.H. et al; SMA Care group. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care// Neuromuscul Disord. 2018 V. 28 (2) P. 103–115

6. Finkel R.S., Mercuri E., Meyer O.H. et al; SMA Care group. Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. // Neuromuscul Disord. 2018 V. 28(3) P. 197-207.

7. Hoy SM. Nusinersen: First Global Approval.Drugs. 2017;77:473-9.

8. Finkel RS, et al. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017;377:1723-1732.

9. Mercuri E, et al. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. 2018;378:625-635.


For citation:Spinal muscular atrophy: unlocking the potential of patients. Clinical review for general practice. 2019; 1: 26-30


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