The effect of coadministration of D156844 and AR42 (REC-2282) on the survival and motor phenotype of mice with spinal muscular atrophy

The effect of coadministration of D156844 and AR42 (REC-2282) on the survival and motor phenotype of mice with spinal muscular atrophy

Abstract Spinal muscular atrophy (SMA) is characterized by degeneration of spinal motor neurons and is a leading genetic cause of pediatric death worldwide. SMA results from the loss of or pathological variant in the survival motor neuron 1 (SMN1) gene. Disease severity is dependent on the number of...

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Bibliographic Details
Main Authors: Ashlee W. Harris, Rod C. Scott, Matthew E. R. Butchbach
Format: Article
Language:English
Published: Nature Portfolio 2025-08-01
Series:Scientific Reports
Subjects:
Motor neuron disease
AR42
2,4-diaminoquinazoline
Spinal muscular atrophy
Preclinical drug trial
Neonatal mouse
Online Access:https://doi.org/10.1038/s41598-025-12194-1
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https://doi.org/10.1038/s41598-025-12194-1

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