Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials
Zanetta C, Nizzardo M, Simone C, Monguzzi E, Bresolin N, Comi GP, Corti S.
Clin Ther. 2014. 1;36(1):128-40.
This article provides a general overview of the main aspects that need to be taken into account to design a more efficient clinical trial and to summarize the most promising molecular trials that are currently in development or are being planned for the treatment of Spinal Muscular Atrophy (SMA).
Keywords: clinical trials, gene therapy, ISIS- SMNRx, olesoxime, oligonucleotides, small molecules, spinal muscular atrophy
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