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Cystic Fibrosis Studies

Since 1998 clinical studies have used Sodium Phenylbutyrate to further the development of a safe and effective treatment for Cystic Fibrosis. Sodium Phenylbutyrate appears to repair the molecular disorder created by the CF gene mutation. According to Scientists, Sodium Phenylbutyrate works as a chaperone for a protein called "The Cystic Fibrosis Transmembrane Conductance Regulator"(CFTR). The gene should ideally "write" instructions for the cell to produce the CFTR proteins. The protein itself is there to create a "channel" in the cell membrane to allow chloride ions to "exit" the cell. These channels through the cell membrane are crucial for the cell to function properly, through which chloride, sodium, calcium, as well as other substances move in and out of the cell, maintaining a proper balance.

Specifically in the lungs, this balance is crucial to maintain mucus of proper composition, to be able to carry away congestion. Ideally the wet and thin mucus is easily removed by the cilia on the outside of the cells. On the other hand, without the proper transport in and out of the cell, the mucus becomes thick and sticky, and accumulates. As a result of this imbalance, bacteria invade the mucus, promoting infections and interfering with breathing.

Because of the gene mutation, the protein is not assembled correctly, and is unable to do its proper function. The cell rejects the disabled CFTR, and, as a result, not enough channels are created for chloride transportation out of the cell. Sodium Phenylbutyrate, on the other hand, flows into the cell through its own channels, accumulates and upsets the balance in the cells.

Copies of clinical trials are available upon request for doctors and medical researchers.

 

 

 

 

 

 

DISCLAIMER:The information presented is intended for educational purposes for health professionals and practitioners, and is obtained from published research. It is not intended to be prescriptive nor to replace the care of a licensed health professional in the diagnosis and treatment of illness.
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