Journal
NEUROLOGICAL SCIENCES
Volume 43, Issue -, Pages 615-624Publisher
SPRINGER-VERLAG ITALIA SRL
DOI: 10.1007/s10072-021-05258-3
Keywords
Antisense oligonucleotides; Gene therapy; Spinal muscular atrophy; Therapy; Nusinersen
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Spinal muscular atrophy (SMA) is a severe disorder of motor neurons, but with three available therapeutic options now, ethical, medical, and financial issues have been raised. Each therapeutic strategy has its weaknesses and strengths, and clinicians need to understand them to optimize clinical care.
Spinal muscular atrophy (SMA) is a severe disorder of motor neurons and the most frequent cause of genetic mortality, due to respiratory complications. We are facing an exciting era with three available therapeutic options in a disease considered incurable for more than a century. However, the availability of effective approaches has raised up ethical, medical, and financial issues that are routinely faced by the SMA community. Each therapeutic strategy has its weaknesses and strengths and clinicians need to know them to optimize clinical care. In this review, the state of the art and the results and challenges of the new SMA therapeutic strategies are highlighted.
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