New medicine for Cystic Fibrosis Treatment

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Rina
Rina's picture
New medicine for Cystic Fibrosis Treatment

The success of the science-medicine synergism and dramatic acceleration in enabling technologies results today, more than even, in translation of scientific “dream” into medical reality. What have motivated me previously as a science student, and is inspiring me today is the translation of our scientific discoveries into improvement in health and quality of life.

As a researcher, who worked on CFTR gene regulation and stability, I am especially excited about the first treatment, available on the market, to target the underlying cause of Cystic Fibrosis. Although KALYDECO (Vertex) is targeted to improve CFTR function in patients with G551D mutation, there are multiple targeted approaches to cystic fibrosis treatment for people with different CFTR mutations.

If you want to know more about this new medicine, please visit Vertex website.
 

sindhu9
sindhu9's picture
Hi Rina,

Hi Rina,

I am just curious to know about the medication you mentioned. Is it specific for nonsense mutations in Cystic fibrosis? I work on a drug PTC 124 to rescue nonsense mutations in Paroxysmal nocturnal hemoglobinuria.

Thanks in advance,
Sindhu

Rina
Rina's picture
I think that VX-770 has

I think that VX-770 has different from PTC124 mechanism of action, since VX-770 acts to create a more effective opening of the CFTR chloride channel already at the cell surface. I advice you to visit the inventor website, if you are looking for more detailed information.

Hope this helps
Rina

andy123
andy123's picture
 Hi

 Hi

This is Useful info. But you have any info regarding this pls visit us 
http://www.accumaximum.com/

thanks.