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Treatment trials may lead to modification of muscular dystrophy diseases

Deborah Y Bradshaw, MDNeurologist Deborah Bradshaw, MD, shares her excitement about two types of disease-modifying treatments that are in clinical trial and could have a profound effect on people who have muscular dystrophy.

“Because we know finally what‘s wrong in the gene, how that translates to an abnormal protein and how the protein may be processed abnormally in the cell, we‘re actually designing drugs that interrupt that pathway and may, literally, change the course of a genetic disease,” said Bradshaw. “It is amazing.”