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Abstract Details

Modeling the Epidemiologic Burden of Duchenne Muscular Dystrophy (DMD) in the United States (US)
Neuromuscular and Clinical Neurophysiology (EMG)
P9 - Poster Session 9 (12:00 PM-1:00 PM)
1-015
To build a model to estimate the projected incidence and age-specific prevalence of DMD in the US.
DMD is a rare X-linked severe myopathy leading to early loss of ambulation and early mortality. Published data estimating the epidemiologic burden of DMD in the US are limited. Age-specific prevalence estimates are available from only one study and limited to diagnosed individuals aged 5-24 years.
Two alternative approaches were explored related to incidence and prevalence of DMD in the US: (1) a systematic literature review (SLR) to identify birth incidence estimates, and (2) numeric calibration using sum-of-squared errors to align model-predicted birth incidence estimates with published age-specific prevalence data. For the modeled birth cohort, published data were identified describing DMD diagnosis and mortality Kaplan-Meier curves. Diagnosis and mortality data were combined via state-transition modeling to estimate age-specific prevalence based on birth incidence. 
Two publications were identified with US-specific birth incidence. Synthesis of Kaplan-Meier curves for disease progression described prevalence initially increasing (reflecting diagnoses between ages 3-12 years), with peak prevalence at 12 years. Following this, disease-specific mortality resulted in declining prevalence by age. When prevalence estimates were modeled beyond ages 5-24, high mortality resulted in rapid decline, with no individuals projected to be alive after age 40. These overall trends were consistently observed across modeled birth incidence estimates; variations of birth incidence only led to variation in magnitude of prevalence at a given age, but not the shape of the prevalence-by-age curve.
While uncertainty remains in specific epidemiological parameters for DMD in the US, synthesizing an epidemiological model provides a framework to extrapolate estimates for age groups that have not previously been described and can be used to calibrate birth incidence estimates. 
Authors/Disclosures

PRESENTER 		No disclosure on file
Karissa Johnston Karissa Johnston has nothing to disclose.
No disclosure on file
No disclosure on file
No disclosure on file