Progress!

Hello blogosphere.  Yes, it’s been awhile.   Seeing that I haven’t posted since the half marathon, perhaps you’ve wondered if I survived.  Well, I did live to tell about it and shall do so soon!  But today, I have some updates to share from the CF world.

Progress in the Lab

Last Monday, May 7 Vertex Pharmaceuticals announced interim results from a Phase 2 clinical trial of Kalydeco™ and VX-809, a CF drug in development.  The results showed a significant improvement in lung function in people with two copies of the most common CF mutation (Delta F508) who received the two drugs in combination.  Both Kalydeco™ and VX-809 are designed to treat the underlying cause of CF.   While these drugs would not be cures for CF, it appears they have the potential to control certain symptoms of CF to a great degree.  Click here to read more about the Phase 2 interim results.

Related articles: http://bit.ly/K76iUX and http://onforb.es/JMy4ah

Progress on the Hill

In this post, I explained that one of the key items on the CFF’s public policy agenda is to get the EXPERRT Act  included in the House and Senate versions of the Prescription Drug User Fee Act (PDUFA).  In short, the EXPERRT Act would require the FDA to ensure that opportunities exist for consultation with external experts on products for CF and other rare diseases.

I am thrilled to share that representatives in both the Senate and the House heard our voices!  The EXPERRT Act has been included in the final versions of PDUFA for both the Senate and the House.  The bills will now proceed to the Senate and House floors for final vote in the near future.   The CFF, my fellow advocates, and I are cautiously optimistic that the core of the EXPERRT act will remain in the final PDUFA bill which will be signed into law.  My State Advocacy co-chair Marissa and I especially appreciate the outreach made by our fellow constituents to Representative Marsha Blackburn.  As a member of the Health Subcommittee of the House’s Energy and Commerce Committee, Congresswoman Blackburn’s support was paramount!

I think this goes to show that we should all follow William James‘ advice:

Act as if what you do makes a difference.  It does. 

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2 Comments

  1. That is wonderful news on the CF front! Congratulations on your success working with Congress. That’s like herding cats while simultaneously nailing jello to the wall.

    Reply
  2. Thank, Jim! And “herding cats” is one of my most favorite metaphors! 🙂

    Reply

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