Rare Disease Day... February 29

Today is Rare Disease Day.

To mark the occasion, PHA partnered with the American Thoracic Society and pulmonary fibrosis groups for a Congressional briefing on pulmonary hypertension and pulmonary fibrosis.  About 40 Congressional offices sent representatives - many because our volunteer advocates contacted them and asked. 

Dr. Susan Shurin, the Acting Director of the National Heart Lung and Blood Institute, hosted and Dr. Seril Erzurum of the Cleveland Clinic and Chair of PHA's Research Committee told the story of the development of PH research and the encouraging story of where she sees it going.  As she spoke of the 9 treatments developed across three unique pathways (endothelin, nitric oxide, prostacyclin) - with two Nobel prizes given for this work - she spoke of new targets and movement towards a cure and the importance of research in this field to other diseases as well.  Her presentation was inspiring but no more so than Nicole Cooper's who spoke movingly of her own journey with PH from diagnosis to her recent operation to remove arterial clots (CTEPH).

When I came back to the office, I read Colleen Brunetti's great blog for Rare Disease Day.  Colleen, a fine writer from the mind and heart reminds us that a disease is not rare if it is in your own body, that treatments are not a cure. 

I encourage you to read Colleen's blog, too.

Pediatric research: The power of one...and a half

It never fails...

One person deciding to take action becomes a driver for a whole community.

Last Friday, PHA board member Steve Van Wormer e-mailed us a public service announcement he had made to support the pediatric research and mentoring that has become an important target for PHA.   We were all very pleased by the the great quality of what he put together...and especially the voice work of Steve's 9 year old son, Lucas.

When we receive a new tool for the community, we always put together a marketing plan to make sure that it is used for the most benefit. 

This time was a little different...

Steve and Lucas' work so obviously filled a need that the small number of people who received it began to move immediately.  It's already being embedded on Facebook pages and pushed out in other ways. 

At its conclusion, it links to the Robyn Barst Pediatric Research and Mentoring Fund which is close to its target for beginning operations.  That Fund page includes information and videos from doctors and parents talking about the importance of pediatric research and creating a body of physicians mentored to capably treat children with PH.

Little more than twenty years ago, PH began to develop as a research and clinical focus.  Now, we are beginning to see - and help drive - that growth among physicians working with children.  While broad PH research remains of critical importance and of benefit to all, pediatric PH research and developing knowledge and understanding of appropriate treatment of children with PH is coming into its time.

What people like Steve and his son, Lucas, are doing in support of the doctors who are building the pediatric field is making that time come faster.  It's a great partnership.

We encourage you to help further circulate the video at the top of this blog.  You can find it here.  Please click on "Like" and you'll be asked if you want it placed on your facebook page...please do. 

Right now, we're working on making e-mails available with an image of the video in them.  I'll update this blog when those are ready

Bruno Kopp: leader, hero, friend...

We still lose too many to this disease...and today we lost another.

Today, it was Bruno Kopp, the leader of the German PH association, PHeV.   He founded the organization in 1996 for the same reason that PHA's founders began ours...to help end isolation.  Five years later, he organized the Rene Baumgart Foundation to raise money for PH research.

Rene Baumgart was Bruno's nephew, a nephew who had lost his mother to PH at the age of 10.  Rene, himself, passed at the age of 19.  I remember Bruno telling me that he became so angry about his nephew's loss, he created the Foundation to forge meaning from what had happened.  You see Rene and his mother weren't Bruno's first loss to this disease.  Fifteen of his family members, including Bruno, had been diagnosed with PH.

In 2006 and 2011, I was honored to stand next to Bruno in Frankfurt and speak at both the 10th and 15th annual Conferences of PHeV and, this year, had hoped that Bruno would be able to make the trip to Orlando in June to PHA's 10th bi-annual Conference.  I am sad that will not be.

During PHeV's 10th Conference, I had been going through some difficult issues.  One evening, Conference attendees were treated to a wonderful caberet performance.  Bruno had asked me to sit next to him.  At one point, he stood and dedicated the next song to me and to PHA.  The song was Carole King's, You've Got a Friend.

Bruno, you were a good man who took the hard things that came into your life and turned them into good for many people.  You were - and are - a hero.

You will not be forgotten...you've got a friend.