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New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges

by 1,2,* and 2
1
Department of Clinical and Experimental Medicine, University of Messina, 98125 Messina, Italy
2
NEuroMuscular Omnicentre (NEMO) Sud Clinical Centre, University Hospital “G. Martino”, 98125 Messina, Italy
*
Author to whom correspondence should be addressed.
J. Clin. Med. 2020, 9(7), 2222; https://doi.org/10.3390/jcm9072222
Received: 3 June 2020 / Revised: 1 July 2020 / Accepted: 10 July 2020 / Published: 13 July 2020
(This article belongs to the Special Issue Innovative Therapies in Neuromuscular Diseases)
Spinal muscular atrophy (SMA) is one of the most common autosomal recessive diseases with progressive weakness of skeletal and respiratory muscles, leading to significant disability. The disorder is caused by mutations in the survival motor neuron 1 (SMN1) gene and a consequent decrease in the SMN protein leading to lower motor neuron degeneration. Recently, Food and Drug Administration (FDA) and European Medical Agency (EMA) approved the antisense oligonucleotide nusinersen, the first SMA disease-modifying treatment and gene replacement therapy by onasemnogene abeparvovec. Encouraging results from phase II and III clinical trials have raised hope that other therapeutic options will enter soon in clinical practice. However, the availability of effective approaches has raised up ethical, medical and financial issues that are routinely faced by the SMA community. This review covers the available data and the new challenges of SMA therapeutic strategies. View Full-Text
Keywords: spinal muscular atrophy; therapy; nusinersen; risdiplam; onasemnogene abeparvovec spinal muscular atrophy; therapy; nusinersen; risdiplam; onasemnogene abeparvovec
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MDPI and ACS Style

Messina, S.; Sframeli, M. New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges. J. Clin. Med. 2020, 9, 2222. https://doi.org/10.3390/jcm9072222

AMA Style

Messina S, Sframeli M. New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges. Journal of Clinical Medicine. 2020; 9(7):2222. https://doi.org/10.3390/jcm9072222

Chicago/Turabian Style

Messina, Sonia, and Maria Sframeli. 2020. "New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges" Journal of Clinical Medicine 9, no. 7: 2222. https://doi.org/10.3390/jcm9072222

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