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Generation 2+ antisense drug

SPINRAZA® (nusinersen) is the first and only approved treatment for all forms of spinal muscular atrophy (SMA) and is approved in more than 50 countries. A new clinical study, DEVOTE, has recently been initiated to assess if higher doses of SPINRAZA can provide even greater efficacy. For more information on the study (NCT04089566), please click here.

Additional information about Spinraza can be found here.

About Spinal Muscular Atrophy (SMA)

Due to a loss of, or defect in the survival motor neuron 1 (SMN1) gene, people with SMA do not produce enough SMN protein, which is critical for the maintenance of motor neurons. SPINRAZA is designed to selectively bind to and alter the splicing of the RNA from the paralog SMN2 gene, to produce full-length, fully functional SMN protein.

Spinal Muscular Atrophy is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness. Ultimately, individuals with the most severe type of SMA can become paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing. People with Type 1 SMA, the most severe life-threatening form, produce very little SMN protein and do not achieve the ability to sit without support or live beyond two years without respiratory support. People with Type 2 and Type 3 produce greater amounts of SMN protein and have less severe, but still life-altering forms of SMA.

Select Publications

  1. Walter M.C. et al.

    (2019) Safety and Treatment Effects of Nusinersen in Longstanding Adult 5q-SMA Type 3 – A Prospective Observational Study. J Neuromuscul Dis. 6(4), 453-465.

  2. Montes J. et al.

    (2019) Nusinersen improves walking distance and reduces fatigue in later-onset spinal muscular atrophy. Muscle Nerve. 60(4), 409-414.

  3. De Vivo D.C. et al.

    (2019) Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study. Neuromuscul Disord. 29(11), 842-856.

  4. Darras B.T. et al.

    (2019) An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials. CNS Drugs. 33(9), 919-932.

  5. Darras B.T. et al.

    (2019) Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies. Neurology. 92(21), e2492-e2506.

  6. Mercuri E. et al.

    (2018) Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. 378(7), 625-635.

  7. Finkel R.S. et al.

    (2017) Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 377(18), 1723-1732.

  8. Chiriboga, C.A. et al.

    (2016) Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy. Neurology. 86(10), 890-897.

  9. Finkel, R.S. et al.

    (2016) Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 388(10063), 3017- 3026.

  10. Finkel, R.S. et al.

     (2014) Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 83(9), 810-817.

  11. Passini, M.A. et al.

    (2011) Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy. Sci Transl Med. 3(72), 72ra18.

  12. Hua, Y. et al.

     (2010) Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev. 24(15), 1634-1644.

  13. Rudnik-Schoneborn, S. et al.

    (2009) Genotype-phenotype studies in infantile spinal muscular atrophy (SMA) type I in Germany: implications for clinical trials and genetic counselling. Clin Genet. 76(2), 168-178.