Trial-ready external controls for gene therapy: the MATCH cohort in maple syrup urine disease

# MATCH Trial: Simulation Code Repository This repository contains the R scripts used to conduct Monte Carlo simulationsfor estimating detectable effect sizes for three outcomes in the MATCH study: - PIPE (%)- Alloisoleucine levels (µmol/L)- Crisis Management Days (CMD) Each script runs a power simulation across a grid of hypothetical treatmentgroup means, generates a power curve, and identifies the smallest (or largest)treatment mean that yields ≥90% empirical power using 10,000 simulated trials. ## Contents - `PIPE_simulation.R`- `Alloisoleucine_simulation.R`- `CMD_simulation.R`- `LICENSE` (MIT License) ## Requirements - R (version 4.2 or higher)- Packages: - ggplot2 - scales ## How to Run Each script is self-contained. Open the desired .R file and run it in RStudio oryour preferred R environment. A CSV file with the power curve results will besaved in the working directory. ## Contact For questions, please contact:Ashlin Rodrigues [arodrigues@clinicforspecialchildren.org]