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ArticleOrphanet journal of rare diseases2014

Polytherapy with a combination of three repurposed drugs (PXT3003) down-regulates Pmp22 over-expression and improves myelination, axonal and functional parameters in models of CMT1A neuropathy.

Ilya Chumakov et al.PubMed ↗Full text ↗Publisher ↗

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The trial

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