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Article

Moving towards treatments for spinal muscular atrophy: hopes and limits

28 Apr 2015

Abstract excerpt

Spinal muscular atrophy (SMA), one of the most frequent and devastating genetic disorders causing neuromuscular degeneration, has reached the forefront of clinical translation. The quite unique genetic situation of SMA patients, who lack functional SMN1 but carry the misspliced SMN2 copy gene, creates the possibility of correcting SMN2 splicing by antisense oligonucleotides or drugs. Both strategies showed...

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PMID: 25920617
DOI: 10.1517/14728214.2015.1041375

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