Natural history study and statistical modeling of disease progression in a preclinical model of myotubular myopathy.

ABSTRACT: Generating reliable preclinical data in animal models of disease is essential in therapy development. Here, we performed statistical analysis and joint longitudinal-survival modeling of the progressive phenotype observed in Mtm1-/y mice, a reliable model for myotubular myopathy. Analysis of historical data was used to generate a model for phenotype progression, which was then confirmed with phenotypic data from a new colony of mice derived via in vitro fertilization in an independent animal house, highlighting the reproducibility of disease phenotype in Mtm1-/y mice. These combined data were used to refine the phenotypic parameters analyzed in these mice and improve the model generated for expected disease progression. The disease progression model was then used to test the therapeutic eff

SUBMITTER: Buono S 

PROVIDER: S-EPMC9346515 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature