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Future Med Chem. 2019 Oct 31. doi: 10.4155/fmc-2019-0239. [Epub ahead of print]

More is needed to complement the available therapies of spinal muscular atrophy.

Author information

1
Department of Biomedical Science, Iowa State University, Ames, IA 50011, USA.

KEYWORDS:

AAV9; ISS-N1; Spinraza; gene therapy; nusinersen; oligonucleotide; spinal muscular atrophy (SMA); survival motor neuron (SMN)

PMID:
31668092
DOI:
10.4155/fmc-2019-0239

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