June 2018. Volume 14. Number 1

The administration of nusinersen improves survival and clinical symptoms in infants with spinal muscular atrophy

 
 
 
 
 
 
 
 
 
 
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AVC | Critically appraised articles

Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377:1723-32.

Reviewers: Ochoa Sangrador C1, Andrés de Llano JM2.
1Servicio de Pediatría. Hospital Virgen de la Concha. Zamora. España.
2Servicio de Pediatría. Complejo Asistencial Universitario de Palencia. Palencia. España.
Correspondence: Carlos Ochoa Sangrador. Email: cochoas2@gmail.com
Reception date: 06/07/2018
Acceptance date: 11/07/2018
Publication date: 18/07/2018

Abstract

Authors’ conclusions: among infants with spinal muscular atrophy, those who received nusinersen were more likely to be alive and have improvements in motor function than those in the control group. Early treatment may be necessary to maximize the benefit of the drug.

Reviewers’ comment: intrathecal nusinersen improves survival free of mechanical ventilation in infants with spinal muscular atrophy. Considering the severity of the disease, both the administration procedure and the high cost of the medication, could be acceptable. Nevertheless, studies that assess the medium and long-term evolution and the cost-benefit ratio of the treatment would be desirable.

How to cite this article

Ochoa Sangrador C, Andrés de Llano JM. La administración de nusinersen mejora la supervivencia y la clínica en los lactantes con atrofia muscular espinal. Evid Pediatr. 2018;14:10.

AVC | Critically appraised articles

Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377:1723-32.

Reviewers: Ochoa Sangrador C1, Andrés de Llano JM2.
1Servicio de Pediatría. Hospital Virgen de la Concha. Zamora. España.
2Servicio de Pediatría. Complejo Asistencial Universitario de Palencia. Palencia. España.
Correspondence: Carlos Ochoa Sangrador. Email: cochoas2@gmail.com
Reception date: 06/07/2018
Acceptance date: 11/07/2018
Publication date: 18/07/2018

How to cite this article

Ochoa Sangrador C, Andrés de Llano JM. La administración de nusinersen mejora la supervivencia y la clínica en los lactantes con atrofia muscular espinal. Evid Pediatr. 2018;14:10.

18/07/2018

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