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Saturday, March 23, 2019

Cystic Fibrosis drug could be made available as early as NEXT MONTH | UK | News | Express.co.uk


BREXIT has invaded the cystic fibrosis space, threatening the health of CF patients in the U.K.

A WONDER drug (ORKAMBI)  could dramatically improve the lives of thousands of Cystic Fibrosis patients may be available to use next month if a funding deadlock is broken, it emerged yesterday.



ORKAMBI has been unavailable for three years since the FDA approved its use in the United States of America. VERTEX developed and distributes Orkambi thanks to a generous donation and a partnership with the Cystic Fibrosis Foundation (USA)

Vertex has been a major player in CF treatment. They also developed the following CF therapeutics
Kalydeco
generic name: ivacaftor 
class: CFTR potentiators
  
10
Orkambi
generic name: ivacaftor/lumacaftor 
class: CFTR combinations
N/A
Symdeko
generic name: ivacaftor/tezacaftor 
class: CFTR combinations
All of these drugs have a specific indication for the F508 chromosomal deletion affecting the CFTR protein on cell membranes which is a gatway for chloride transfer.

A description of Cystic Fibrosis was noted in the middle ages.

“Woe is the child who tastes salty from a kiss on the brow, for he is cursed and soon must die”.



Cystic Fibrosis drug could be made available as early as NEXT MONTH | UK | News | Express.co.uk:

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