Making Sense of 6MWT Variability: Developing a Disease Progression Model for DMD

Lora Hamuro

Duchenne Muscular Dystrophy (DMD) is a life-threatening, sex-linked, pediatric rare disease, primarily affecting boys. It is characterized by progressive muscle degeneration, weakness, and eventually functional loss. DMD is caused by a mutation in the dystrophin gene, a protein needed to maintain muscle integrity, and for improving signaling and growth in differentiation of the muscle tissue. […]

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Topics: PK/PD Modeling & Simulation