[Editorial] Spinal muscular atrophy, pediatric virology and gene therapy: A challenge of modern weakness and hope
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- Published online on: February 22, 2018 https://doi.org/10.3892/etm.2018.5883
- Pages: 3671-3672
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Copyright: © Darras et al. This is an open access article distributed under the terms of Creative Commons Attribution License.
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Abstract
Spinal muscular atrophy (SMA) type I is a devastating diagnosis for ‘the smiling infant, who progressively develops weakness, hypotonia and respiratory distress, related to respiratory failure and life-threatening infections caused by common pathogens’ (1). These pathogens include bacteria and various viruses, such as respiratory syncytial virus (RSV) and seasonal influenza viruses. Despite the fact that there is, currently, no cure [iasis (ἴασις) in Greek] for SMA type I, it does not mean that that there are no therapeutic options [therapia (θεραπεία) in Greek] and hope (2,3). There are several paths the families of the affected children can follow, depending on the country they live in and the medical specialized services that can be offered to them. These options aim at managing symptoms, reducing the complications of muscle weakness and maintaining optimal quality of life. One of the options is non-invasive ventilation, which ‘when combined with nutritional support, can indeed prolong survival’ (1); the use of invasive ventilation remains controversial. In several countries worldwide, it is currently possible to access a new therapeutic intervention for SMA, a drug named Nusinersen, also known as Spinraza™. The child's parents/guardians and the child's medical team are expected to come to a decision as to its potential benefit for their child with SMA type I. In the meantime, there is ongoing research in leading worldwide centers, such as the Boston Children's Hospital in Boston (MA, USA), with hope that this research will lead to other treatments for SMA (4).