| Title: |
Mathematical modeling of the progression of Duchenne Muscular Dystrophy in San Diego in an era of allele specific oligonucleotide therapy |
| Authors: |
Kingsmore, Stephen; Baun, Dominic; Tobin, Laura; Nguyen, Hung; Arenchild, Madison; Juarez, Edwin; kiernan, elizabeth; Lesser, Daniel; Wong, Emily; Prilutsky, Daria; Zach, Neta; Han, Steve; Diogo, Dorothee; Szalma, Sandor; Laverty, Chamindra |
| Publisher Information: |
Springer Science and Business Media LLC |
| Publication Year: |
2024 |
| Description: |
Duchenne muscular dystrophy (DMD) is a relatively common, severe, progressive, X-linked muscle disorder caused by pathogenic variation in the dystrophin gene. Affected status can be determined at birth, 3-5 years before symptom onset. As effective corticosteroid and genetic therapies are becoming available for DMD, predictive models of DMD progression are needed to guide individualized care of patients and clinical trials of new treatments. We developed a multivariable, dynamical model of DMD progression, as measured by percent of predicted FVC (FVC%), through a longitudinal natural history study of 113 boys from a single center in San Diego County between 2008 – 2023. A mixed effect linear regression model of FVC% had marginal and conditional r 2 values of 0.54 and y of 0.83 with five factors (Hispanic ethnicity, B= -10.0, 95% CI -16.2 to -3.89, p=.002; age at symptom onset, B= -2.4, 95% CI -3.6 to -1.3; p |
| Document Type: |
other/unknown material |
| Language: |
unknown |
| DOI: |
10.21203/rs.3.rs-4049883/v1 |
| Availability: |
https://doi.org/10.21203/rs.3.rs-4049883/v1; https://www.researchsquare.com/article/rs-4049883/v1; https://www.researchsquare.com/article/rs-4049883/v1.html |
| Rights: |
https://creativecommons.org/licenses/by/4.0/ |
| Accession Number: |
edsbas.4B872605 |
| Database: |
BASE |