Expanding the Duchenne Therapeutic Opportunities by Targeting Myostatin - PPMD Community

Expanding the Duchenne Therapeutic Opportunities by Targeting Myostatin - PPMD Community: "We all understand that Duchenne muscular dystrophy is characterized by muscle wasting and associated loss of function and that there are considerable efforts underway to develop drugs and biologics (cell and gene therapy) to address the primary problem in Duchenne—the absence of dystrophin. Restoring dystrophin or replacing dystrophin with replacement protein are considered foundational therapies.
 
That said, the Duchenne community is well aware of the need for combination therapies. Combinations would include compounds that target what is referred to as the ‘downstream pathology’ or the changes that occur because dystrophin is absent. This includes anti-inflammatories, anti-fibrotics, factors that control muscle regeneration and fiber size, compounds that improve circulation to muscle, and compounds that improve mitochondrial function (mitochondria are considered the powerhouses of cells). We are all hopeful that by combining several of these targeted therapies, we could end Duchenne, stop progression for every individual. This is the dream of precision or personalized medicine. The right drug, at the right time, in the right dose for the right person. It requires planning and it will require combinations."



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Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner

Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner: "Statins are widely known for their use in improving cardiovascular health through lowering blood cholesterol levels. What prompted you to study their use in Duchenne muscular dystrophy (DMD)?"



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