HAEA TODAY 

        Summer  2009
            Newsletter 
 
A Message From Your HAEA President
 
Dear HAE Friends,

I hope all are having an enjoyable summer. Trends in the HAE world continue to be very positive!

Before I discuss the progress made in bringing new HAE medicines to the US, I want to stress that the HAE Association is here to help you.  Please do not hesitate to call your Regional Patient Services Representative (see the list and phone numbers at the bottom of this newsletter) with any questions or concerns. The HAE Association is built on a foundation of caring and compassion and every staff member is dedicated to doing whatever is necessary to help our fellow HAE friends.

Regarding progress made with HAE treatments, the Cinryze brand of C1 inhibitor concentrate is now commercially available for preventing HAE attacks, and FDA will soon decide whether or not to approve another brand of C1 inhibitor--Berinert--for treating acute HAE attacks. The FDA will also make a decision on the Dyax Corporation's DX-88 product in December, and Jerini is initiating another clinical trial for their drug, Firazyr.

As noted below, we are planning another National Patient Conference for October, so please mark your calendars. We are working hard to ensure that the 2009 gathering will be the best HAE patient meeting ever!

Warmest regards to all,
Tony Castaldo
President, US HAEA  
US HAEA 2009 National Patient Conference to Be Held in Orlando, Florida !
 
 Mark your calendars for Oct. 23-
 23- 25, 2009 and start planning to 
 attend what promises to be a most
 informative and fun conference!
 
 Attendees will have a chance to   
 meet other HAE friends and get the most up to date information on HAE treaments and research!
 
Please stay tuned for more detailed information, but be sure to keep these dates open!
Introducing the New US HAEA Scientific Registry : Patient Driven Research toward a Cure!
 
beakers
 
The US Herediary Angioedema Association is proud to announce an exciting new endeavor that is squarely aimed at directing research toward a cure for HAE. 
 
We will soon be asking each and every HAE friend to sign up and provide information for our Scientific Registry!  Data is the raw material that researchers need to find better treatments and a cure.
 
Watch for more information on how you can participate in this important project!
US HAE  Association Research Featured at HAEA Scientific Meeting in Europe
 
research
 US HAE Association-sponsored
 research was prominent during
 a recent C1 Inhibitor Workshop  
 conducted in Budapest,  
 Hungary in late May. 

The first presentation included an overview of the HAEA's  Burden of Illness Study which drew upon quality of life and other data gathered from 475 HAE friends.  The study clearly demonstrates that HAE imposes substantial financial and psychological burdens for HAE patients in just about every facet of life.
 
US HAEA Medical Advisor Professor Bruce Zuraw,  presented two other HAEA sponsored studies that were co-authored by HAEA President Anthony Castaldo and HAEA Director of Patient Services Donna Davis.:
      1.  The safety and benefits of self (home) admin-
           istered C1 Inhibitor; and
      2.  The effectiveness, safety and side effects of
           anabolic steroids for treating HAE
 
With respect to the C1 Inhibitor self-infusion study, Professor Zuraw reported that home C1INH therapy can be done safely and results in a significantly higher quality of life for HAE patients.
 
The anabolic steroid study was the largest scale HAE research project ever accomplished.  731 HAE friends responded to a sophisticated online survey that measured the effectivenss and side effects of steroids.  The study revealed that anabolic steroids are often effective for preventing HAE attacks, but the vast majorityof patients believe the medicine's side effects outweigh the benefit and would consider other forms of therapy as they become available.
 
Summaries (abstracts) of these studies appear on the HAEA website   www.haea.org
In This Issue
 
 
 A Message   
 From  Your
 HAEA President
 
 US HAEA 2009
 National
 Patient  
 Conference
 to Be Held in
 Orlando, 
 Florida!
 
 Introducing the 
 New US HAEA
 Scientific
 Registry:
 Patient Driven
 Research
 Toward a
 Cure !
  
 US HAE
 Association 
 Research
 Featured at 
 HAE Scientific
 Meeting in
 Europe
 
 
 Newly 
 Approved
 HAE Therapy
 
 FDA Decisions
 Pending on
 Other HAE
 Medications 
 
 
 Clinical Trial
 Opportunity 
 
 
  Meet your
 HAEA
 Advocates
Also in this Issue
 Your Patient
 Services
 Representatives
 by Region
 
Newly Approved HAE Therapy
 
pharmas  The Cinryze brand of C1 Inhibitor - a medicine that boasts a 30-year
  track record of remarkable safety and effectiveness - is now FDA
  approved and available to prevent attacks of HAE.
 
  We are pleased to report that the FDA has recently approved Cinryze
  for home infusion. 
 
  Please contact an HAE Association Patient Services Representative for more information about this exciting new option for preventing HAE attacks.
FDA Decisions Pending on Other HAE Medicines
 
clinical   The FDA is currently reviewing CSL Behring's Berinert brand of C1
   Inhibitor for treating HAE attacks.  A decision for 
   approval/disapproval willl be made in September. 
 
   The DYAX Corporation announced that the FDA will decide on
   whether to approve the company's medicine - Dx-88 - for treating
   HAE attacks.  If approved, the drug (administered by three
   subcutaneous injections) will be available at medical facilities.  In response to questions from the FDA, the company submitted a drug safety plan and information on manufacturing controls. The FDA will announce an approval/diapproval decision on December 1, 2009.
Clinical Trial Opportunity
 
clinical2  Jerini/Shire US is recruiting HAE patients for a clinical trial
  testing its medicine (Firazyr) - a treatment for HAE attacks.
  Firazyr is administered as one subcutaneous injection.  
 
  HAE friends interested in participating in  this trial  should
  contact  your HAEA Patient Services Representative (see list
  below).
Meet your HAE Advocates
 
donna2
  Donna Davis - Director of Patient Services and Clinical Programs
 
  Donna is responsible for leading and overseeing the important 
  work accomplished by the HAEA's Patient Services group. She
  began focusing on HAE in 1995 when her son Kris was diagnosed
  with an extremely severe case of HAE at age 5.  "I was
  devastated by the relentless and horrific HAE attacks my son was
  suffering, and was determined to find a way to help him and
  others who suffer from frequent, debilitating swelling."  Donna
  joined forces with other HAE advocates she met through the HAE email group and was part of the small group that formed the HAE Association. She was the HAEA's first employee and has worked her way up to being appointed  leader of the HAEA's Patient Services Group. 
 
According to Donna, "My job has provided me the opportunity to speak with hundreds of HAE friends throughout the country, and I am continually shocked at what our patient community must endure--decades of misdiagnosis, uninformed treatment and unneccesary surgeries accompanied by what, at times can only be described as cruel and dangerous hospital care." 
 
Donna adds, "Our Patient Services Group exists to serve HAE patients and we aim to do whatever possible to help HAE friends lead a better life."  She believes that the recently approved Cinryze C1 inhibitor product will have a dramatic impact on the health of US HAE patients and provides the first bona fide alternative to toxic anabolic steroids. Donna encourages all HAE friends interested in pursuing Cinryze therapy to contact her or any of the HAEA Patient Services Representatives.  She is also very excited about the prospects for other HAE medicines that are in the FDA approval pipeline.
   

Michelle
  Michelle Williamson - Assistant Director of Patient Services
 
  Michelle Williamson has an extremely severe case of HAE and,
  untreated, experiences swelling attacks every 48 to 72 hours.
  Because she has survived dozens of intubations and a
  tracheotomy, Michelle truly understands the burden endured by
  many HAE friends. She got involved with the HAEA by posting to
  the List Serve email group and then realized that she wanted to
  be part of the fight to bring new and better treatments for the HAE
  community.  Michelle joined the HAEA in January 2006 and was
  promoted to Assistant Director of Patient Services last year.
 
Michelle said, "I am dedicated to doing whatever I can to make sure HAE friends avoid the life of pain, suffering, and dismay that I had to endure before the HAEA helped me get involved in a clinical trial. The Cinryze clinical trial gave me back my life, and I want to make sure all HAE friends know this incredible therapy is now commercially available."  She is passionate about providing HAE friends with information about Cinryze and other new HAE therapies that are pending FDA approval. 
 
According to Michelle, "One of my goals is to make sure that all HAE friends know that the HAEA has a wide variety of services available to help people in our community lead a normal life.  I am determined to continue reaching out and making sure everyone in our community is part of our network, and has the chance to experience, first hand, the HAEA's "Culture of Caring".
Your Patient Services Representatives by Region
 
phone      Region-1  Michelle Williamson     (972) 814-5205      
      michellewilliamson@haea.org 
      (Region 1 includes-Arizona, Colorado, Washington DC,       
      Kansas, Kentucky, Maine, Nebraska, New Mexico, Nevada
      and Texas)   

Region-2  Lois Perry         (559) 259-0572       
loisperry@haea.org 
(Region 2 includes-California, Iowa, Illinois, Indiana, New Hampshire, Oregon, Tennessee,  Utah, Virginia and West Virginia)
 
Region-3  Donna Davis      (808) 216-1029      
donna-davis@haea.org 
(Region 3 included-Alaska, Hawaii, Idaho, Minnesota, Missouri, Montana, Rhode Island, Wisconsin and Wyoming)
 
Region-4  Sally Urbaniak   (904) 826-6700      
surbaniak@haea.org 
(Region 4 includes-Connecticut, Delaware, Florida, Massachusetts, New Jersey, New York, North Dakota , Ohio, Pennsylvania, Vermont and Washington)
 
Region-5   Jenny Barnes    (252) 585-0763      
jennybarnes@haea.org 
(Region 5 includes-Alabama, Arkansas, Georgia, Louisiana, Maryland, Michigan, Mississippi, North Carolina, Oklahoma, South Carolina and South Dakota) 
 
Patient Services/Clinical Programs Representative    John Williamson   (972) 984-0621
john@haea.org 
The US HAE Association publishes its newsletters to ensure everyone in our HAE family is informed about important clinical developments and all HAE Association activities.  We hope you have enjoyed this issue!
 

The United States Hereditary Angioedema Association
Seven Waterfront Plaza
500 Ala Moana Blvd., Suite 400
Honolulu, HI   96813
 
Toll free phone:  (866) 798-5598