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In many diseases, the development of fibrosis in muscle, or any tissue, is the result of a reactive, or faulty tissue repair process involving many factors.


In Duchenne, fibrosis is present in skeletal muscles at a very early age and is one of the major factors contributing to the loss of muscle strength and function. Fibrosis develops in Duchenne after multiple cycles of muscle degeneration (injury), inflammation, and regeneration (repair).


One of the factors involved in muscle healing and regeneration, and the eventual development of fibrosis, is connective tissue growth factor (CTGF). Potentially, blocking the action of CTGF with anti-connective tissue growth factor might reduce fibrosis (i.e., have an anti-fibrotic effect), preserving muscle structure, strength and function.

 

What does Idiopathic Pulmonary Fibrosis have to do with Duchenne? 

 

Ideopathic Pulmonary Fibrosis (IPF) is a disease causing the tissues in the lungs to become stiff and fibrotic. Recently, FibroGen announced positive topline results from their Phase 2 randomized, double-blind, placebo controlled study of Pamrevlumab – an anti-connective tissue growth factor (anti-fibrotic) in IPF. CGTF is critical for the development of fibrosis in both IPF and in Duchenne.

While Duchenne and IPF are different diseases, the cellular mechanisms that promote fibrosis are the same, so it is reasonable to think that if Pamrevlumab worked in IPF, it might work in Duchenne. In addition, FibroGen’s recent press release suggested that Pamrevlumab has a good safety profile, critical to our thinking around benefit and risk.

 

Pamrevlumab & Duchenne

 

This is good news for Duchenne. The FibroGen study, MissionDMD, a Phase 2, open label study for non-ambulatory patients, continues to recruit. Individuals enrolled in the study will receive 35mg/kg pamrevlumab (FG-3019) every two weeks, by IV infusion for up to 104 weeks. Click here for more information on trial criteria. Always discuss questions or your interest in trial participation directly with your care provider.

 

Positive news from another disease that develops fibrosis which limits the ability to breathe, has the potential to have a similar impact on fibrosis in Duchenne. And with luck, completion of the study and analysis will lead to one more piece of the puzzle, one more therapy we include in the combination of therapies that will End Duchenne.

FibroGen is an educational partner of PPMD.

 

 

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