SMA Treatment May Be More Effective by Altering Underlying Molecule

SMA Treatment May Be More Effective by Altering Underlying Molecule

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SMA Treatment May Be More Effective by Altering Underlying Molecule

Biochemical modifications made to antisense oligonucleotides — molecules that are the basis of an existing therapy for spinal muscular atrophy (SMA) — may improve this and similar treatments, new research suggests. More work, which is underway, is needed as the biochemical changes seen to possibly offer an advantage were more effective in lab than in animal studies, its scientists said. These findings were in the study, “Mesyl Phosphoramidate Oligonucleotides as Potential Splice-Switching Agents: Impact of Backbone Structure…

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