Toxicologic pathology
Risdiplam: A Small Molecule mRNA Splice Modifier Approved to Treat SMA Disease.
Lutz Müller, Björn Jacobsen, Alexander Nürnberg
Published: 202510.1177/01926233251392863
Abstract
Risdiplam is the only approved small molecule mRNA splice modifier to date. Over more than five years since its first approval, thousands of patients with spinal muscular atrophy have benefited from risdiplam therapy. Yet, the nonclinical development…
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