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J. Clin. Med. 2018, 7(9), 251; https://doi.org/10.3390/jcm7090251

New Directions for SMA Therapy

1
Department of Clinical and Experimental Medicine, University of Messina, 98100 Messina, Italy
2
NEuroMuscular Omnicentre (NEMO) Sud Clinical Centre, University Hospital “G. Martino”, 98125 Messina, Italy
Received: 19 July 2018 / Revised: 24 August 2018 / Accepted: 25 August 2018 / Published: 31 August 2018
(This article belongs to the Special Issue Hereditary Neuromuscular Diseases)
Full-Text   |   PDF [221 KB, uploaded 31 August 2018]

Abstract

Spinal muscular atrophy (SMA) is a severe disorder of motor neurons and the most frequent genetic cause of mortality in childhood, due to respiratory complications. The disease occurs due to mutations in the survival motor neuron 1 (SMN1) gene that leads to a reduction in the SMN protein, causing degeneration of lower motor neurons, muscle weakness and atrophy. Recently, the Food and Drug Administration (FDA) and the European Medical Agency (EMA) approved the antisense oligonucleotide nusinersen, the first disease-modifying treatment for SMA. Encouraging results from SMN1 gene therapy studies have raised hope for other therapeutic approaches that might arise in the coming years. However, nusinersen licensing has created ethical, medical, and financial implications that will need to be addressed. In this review, the history and challenges of the new SMA therapeutic strategies are highlighted. View Full-Text
Keywords: antisense oligonucleotides; gene therapy; spinal muscular atrophy; therapy; nusinersen antisense oligonucleotides; gene therapy; spinal muscular atrophy; therapy; nusinersen
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).
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Messina, S. New Directions for SMA Therapy. J. Clin. Med. 2018, 7, 251.

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