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

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. […]

Author(s): Lora Hamuro
Therapeutic Area: Pediatrics, Rare/Orphan Disease
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Level C IVIVC: What Is It, and When Do You Use It?

In vitro in vivo correlation (IVIVC) links in vitro drug data to in vivo performance. Regarding in vivo performance, IVIVC can predict either the in vivo plasma time-concentration curve or in vivo pharmacokinetic (PK) parameters like Cmax and AUC. In this blog, I will discuss Level C IVIVC, and when you should use this approach. […]

Author(s): Jean-Michel Cardot
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