RE:RE:My guess is that PBI 4050 will treat cystic fibrosisI'm taking a few hours off this morning.
I'm trying to link the mechanism of action of PBI-4050 with disease relatade to an inflammory condition and anything involving TGF.
I didn't find how excactly cystic fibrosis is working, apart a mutation on a protein that I never seen involve in the MOA of 4050.
However, knowing that PBI-4050 is working on several organs with fibrosis, and keeping in mind that the skin is an organ, I see as a possibility :
Systemic sclerosis.
A little read about it.
Systemic Sclerosis
Systemic Sclerosis (Scleroderma) is a chronic connective tissue disease generally classified as one of the autoimmune rheumatic diseases with an unclear etiology. Scleroderma is found it two forms: limited and diffuse, with the diffuse form being most severe, affecting around 50,000 people in the United States. About 80% of those affected by scleroderma are women with an onset typically in her mid-life.
In diffuse systemic sclerosis, the body's immune system attacks and damages the skin causing it to thicken rapidly over a large area and may eventually involve the esophagus, gastrointestinal tract, lungs, kidneys, heart and other internal organs. It can also affect blood vessels, muscles and joints. There is currently no cure or effective therapy for scleroderma with pulmonary fibrosis being the most common cause of mortality.
Inflammation is the driving force behind the disease symptoms, leading to progressive fibrosis and eventual mortality. In particular, the pro-inflammatory and pro-fibrotic cytokine TGF-beta has been identified as a key player in the progression of the disease and is considered an important target for therapy.