August 8, 2020 /

In the Pipeline: Downstream Targets

When there isn’t enough dystrophin in the body, the muscle is damaged and the body naturally tries to repair this damage through muscle regeneration. This continual state of muscle degeneration and regeneration produces other effects such as fibrosis, inflammation, calcium imbalance, muscle wasting, cellular energy depletion (mitochondria), and cardiac dysfunction. These are called downstream effects because they happen due to the lack of dystrophin. This panel will feature companies developing therapeutic strategies aimed at addressing downstream targets focused on mitochondria and the heart.

Astellas Pharma (Gerard Marek MD PhD, Executive Medical Director)
Santhera Pharmaceuticals (Kristina Nygren, MD, Chief Medical Officer)
Cumberland Pharmaceuticals (Larry Markham, MD)
Epirium Bio (Ransi Somaratne, M.D, CMO)

Followed by Q &A

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