Small Molecules in Development for the Treatment of Spinal Muscular Atrophy

Alyssa N. Calder, Elliot J. Androphy, Kevin J. Hodgetts

Research output: Contribution to journalReview article

34 Scopus citations


Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease resulting from pathologically low levels of survival motor neuron (SMN) protein. The majority of mRNA from the SMN2 allele undergoes alternative splicing and excludes critical codons, causing an SMN protein deficiency. While there is currently no FDA-approved treatment for SMA, early therapeutic efforts have focused on testing repurposed drugs such as phenylbutyrate (2), valproic acid (3), riluzole (6), hydroxyurea (7), and albuterol (9), none of which has demonstrated clinical effectiveness. More recently, clinical trials have focused on novel small-molecule compounds identified from high-throughput screening and medicinal chemistry optimization such as olesoxime (11), CK-2127107, RG7800, LMI070, and RG3039 (17). In this paper, we review both repurposed drugs and small-molecule compounds discovered following medicinal chemistry optimization for the potential treatment of SMA.

Original languageEnglish (US)
Pages (from-to)10067-10083
Number of pages17
JournalJournal of Medicinal Chemistry
Issue number22
StatePublished - Nov 23 2016

ASJC Scopus subject areas

  • Molecular Medicine
  • Drug Discovery

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