Duchenne Israel held its first conference on Tuesday evening. Professor Yoram Nevo, the leading pediatric neurologist and CINRG director opened the meeting with a discussion about his efforts to combine Losartin and Copaxone as a potential substitute for steroids. He has data on each compound and will now test the combination in the mdx mouse. 60+ family members, doctors, researchers, and healthcare professionals attended the meeting. The room was filled – over 60 family members, doctors, researchers and healthcare professionals gathered together for this meeting. The room was filled with excitement, with community, with hope. This is my first trip to Israel and as I stood on that podium, I saw the diversity in the room –different cultures, different belief systems – united in the universal language called Duchenne muscular dystrophy. It was exactly right. We should be together, working side by side.
This afternoon my wonderful hosts took me to the Wailing Wall and to the tunnels, the excavation site to expose the entire Western Wall. Our tour guide provided the historical prospective. We looked back in order to look forward. As we climbed down into the tunnels, the guide explained we were in the central part of the Western Wall, the place thought to be nearest to the Holy of Holy places. We stopped there in prayer, wrote names of the young men we lost this year and placed the notes into the wall.
We walked down the Via Dolorosa, the path Jesus carried His cross. We touched the stone where his body was placed after He died. We again prayed for all of our sons and daughters. We prayed for all of us. Israel. Faith. Hope.
Today is the last day to vote for PPMD in the Chase Community Giving challenge on Facebook. I have voted for Duchenne, for my own sons and for yours. We may not win the $1 million grand prize, but we must vote so that the Facebook universe understands how important our fight is. And so that our children understand how precious we think they are. This contest was never about a big cash prize (sure that would be great, we'll never turn down $1 million!) - it's about awareness.
I'm on the other side of the world from most of you and the faces of Duchenne are just as beautiful here as they are back in the States. Your vote for PPMD and Darius Goes West (another Duchenne specific organization that shares our mission to end Duchenne) is not just for your son or your friend's son, but for the young people all around this world living with Duchenne.
Friday, January 22, 2010
Tuesday, January 19, 2010
The Marathons of Life
On January 9, I ran the Disney ½ marathon in Orlando. Well, ‘run’ may be a loose term, I walked, but I walked fast enough to complete the race in the required time, crossing the finish line, greeted by a wonderful volunteer holding the coveted Donald Duck medal. It was my third marathon.
I started walk/running in 1984, just after the word Duchenne entered my vocabulary. I was running for something, my own private marathon. I walked for my sons. I wanted to take steps for them, wanted to make up for the steps they would not take and wanted to take steps for others as well. I didn’t calculate how many steps that might take, but I knew that after taking one step, I would be able to continue the momentum.
And now, my private marathons are public, are part of something much larger than myself, something that belongs to all of us. I’m a believer.
55,000 people were running those races in Disney. 55,000 running for something or someone they care about, something or someone they are committed to. 55,000 people committed to succeed.
There is another marathon, but this time, it does not involve running. This uses no energy and with one single click on the computer, will help change the world for 250,000 boys with Duchenne and a few girls and millions of people who love them.
PPMD recently was announced as a finalist in the Chase Community Giving challenge on Facebook. Over 500,000 organizations competed for 100 finalist slots and a $25,000 prize. You voted, and we made it! Now these 100 organizations will compete for five $100,000 prizes and one grand prize of $1 million.
So we need you to vote. And we need you to tell everyone you know on Facebook to vote. And we need you to get everyone who is not on Facebook, to join Facebook, and then vote. The power your one vote could have is incredible. That vote could mean $1 million for Duchenne research. That vote could buy us time.
1 vote. 1 minute. $1 million for Duchenne research.
And if you have 5 minutes we have a list of super easy, super effective ways you can reach as many people on Facebook and have them cast their vote for PPMD. We need all the support on Facebook we can get. Every vote counts!
I am not a seasoned athlete, but I am always able to finish because of the energy of those around me. That energy, YOUR energy, will help us win $1 million to be used in the fight to end Duchenne.
I hope you'll vote today. No training required.
I started walk/running in 1984, just after the word Duchenne entered my vocabulary. I was running for something, my own private marathon. I walked for my sons. I wanted to take steps for them, wanted to make up for the steps they would not take and wanted to take steps for others as well. I didn’t calculate how many steps that might take, but I knew that after taking one step, I would be able to continue the momentum.
And now, my private marathons are public, are part of something much larger than myself, something that belongs to all of us. I’m a believer.
55,000 people were running those races in Disney. 55,000 running for something or someone they care about, something or someone they are committed to. 55,000 people committed to succeed.
There is another marathon, but this time, it does not involve running. This uses no energy and with one single click on the computer, will help change the world for 250,000 boys with Duchenne and a few girls and millions of people who love them.
PPMD recently was announced as a finalist in the Chase Community Giving challenge on Facebook. Over 500,000 organizations competed for 100 finalist slots and a $25,000 prize. You voted, and we made it! Now these 100 organizations will compete for five $100,000 prizes and one grand prize of $1 million.
So we need you to vote. And we need you to tell everyone you know on Facebook to vote. And we need you to get everyone who is not on Facebook, to join Facebook, and then vote. The power your one vote could have is incredible. That vote could mean $1 million for Duchenne research. That vote could buy us time.
1 vote. 1 minute. $1 million for Duchenne research.
And if you have 5 minutes we have a list of super easy, super effective ways you can reach as many people on Facebook and have them cast their vote for PPMD. We need all the support on Facebook we can get. Every vote counts!
I am not a seasoned athlete, but I am always able to finish because of the energy of those around me. That energy, YOUR energy, will help us win $1 million to be used in the fight to end Duchenne.
I hope you'll vote today. No training required.
Thursday, January 14, 2010
Tuesday, January 5, 2010
Aligning the Stars
PPMD is growing. We have an amazing Board, an equally amazing staff (commonly referred to as the Cult), and a wonderful community. So what should be the logical next step? This has been our conversation for the last year. It is easy right? Add staff. But as you begin to think of who that person might be, you start to develop a wish list, naming all of the qualities/skills you are hoping to find in a person. And then there is Duchenne. How do you explain Duchenne to someone who has no experience? It is not an easy subject and certainly not simple. This means, the wish list expands to find someone with a passion for Duchenne, someone who understands the need to buy TIME, someone with significant experience and knowledge about research, someone who ‘gets it’, understands the full impact of the diagnosis. Someone like Sharon Hesterlee. Boy, if only we had a Sharon Hesterlee.
Sundays are not my favorite days. People rest. I fidget. On Sunday some months ago, I received a message from Sharon Hesterlee. She wanted to talk. The thing is, I like to talk with Sharon Hesterlee. I met her long ago, when she joined the MDA to lead their research investments. She is smart, well-versed, knows the field and the players. She understands the investments and is a well-respected leader in the field. She knows the obstacles, the bottlenecks, and the politics. And, she has been around this community for over 10 years. She understands the value of developing collaborations, recognizes the need to leverage money, and understands all of the moving parts. This Sunday was looking up!
I called. We discussed a range of issues, most specifically her interest in focusing on a single disease – Duchenne. You might imagine, I was smiling. And thrilled when she said she would like to work for PPMD. I called John Killian (our Chairman) and he connected with the Board. Sharon flew into NJ and met the staff. Sometimes you just know things and this felt exactly right.
After trying my best not to shout this news from the rooftops for the last few months, I’m thrilled to be able to say – Sharon Hesterlee works for PPMD. She is Senior Director of Research and Advocacy.
Some days, the stars line up just right. Happy 2010.
Sundays are not my favorite days. People rest. I fidget. On Sunday some months ago, I received a message from Sharon Hesterlee. She wanted to talk. The thing is, I like to talk with Sharon Hesterlee. I met her long ago, when she joined the MDA to lead their research investments. She is smart, well-versed, knows the field and the players. She understands the investments and is a well-respected leader in the field. She knows the obstacles, the bottlenecks, and the politics. And, she has been around this community for over 10 years. She understands the value of developing collaborations, recognizes the need to leverage money, and understands all of the moving parts. This Sunday was looking up!
I called. We discussed a range of issues, most specifically her interest in focusing on a single disease – Duchenne. You might imagine, I was smiling. And thrilled when she said she would like to work for PPMD. I called John Killian (our Chairman) and he connected with the Board. Sharon flew into NJ and met the staff. Sometimes you just know things and this felt exactly right.
After trying my best not to shout this news from the rooftops for the last few months, I’m thrilled to be able to say – Sharon Hesterlee works for PPMD. She is Senior Director of Research and Advocacy.
Some days, the stars line up just right. Happy 2010.
Holly PA
What’s new at PPMD? Holly Peay (pronounced P-A). Well, not exactly new, but new in that Holly has joined the staff of PPMD! We first met Holly when working on DuchenneConnect, the patient self-report registry. She came highly recommended as a genetic counselor, an educator, and an all-around, really smart woman. Her task was to create educational materials for families and for providers – educational information about Duchenne, about genetic testing, about care, about research, about clinical trials; and present it in such as way, that it would be comprehensive and understandable, even if the information was really fresh and even if tears might get in the way of your reading from time to time. She did it! She made very difficult information understandable.
Through DuchenneConnect and the PPMD Community site, we heard stories. Stories about delays in diagnosis…months…years at times. We thought about what we might do to help. We had some experience.
We submitted a grant to the Center for Disease Control (CDC) and were granted a cooperative agreement. The goal of the grant is to educate, to raise awareness about muscle weakness, and to impact the diagnostic odyssey. Our initial experience was in Mississippi. We developed partnerships with healthcare professionals likely to see children in the early years of life (ages birth to 5 years). We wanted to understand what happens when mom, dad, gram, or anyone really, expressed concern about a child’s muscle strength. What words did they use? What happened when they raised concern with the doctor (nurse, physician’s assistant, healthcare professional, teacher).
And we learned that some of the time, concerns are dismissed. We learned that some doctors/healthcare professionals are not exactly sure what they are to look for. We learned that some of the time, referrals to specialists are difficult with long waiting times. We learned that sometimes the response is to send the child to a PT or put them into an exercise program to improve strength. And the diagnostic odyssey continues. Typically mom, dad, gram’s worry is relieved for only a short time because they see something is still not quite right.
With this current cooperative agreement, we are expanding our horizons. Our goal is to educate healthcare professionals and this time, not in just a specific state but rather now through the professional societies. There are too many acronyms to list – but professional societies of every healthcare professional group you can think about – pediatrics, family doctors, physician’s assistants, nurse practitioners, nurses, physical therapy…you get the gist.
We have expanded this effort to include ‘primary muscle disorders of childhood’ – because the message will have greater impact. And Duchenne is the most common
We have developed common messages:
• Watch them walk, run, climb (in the confines of an exam room, sometimes it is difficult to really see what mom, dad, gram is talking about)
• Developmental Delay, include a CK (you already know this one)
• YOU CAN ALWAYS DO SOMETHING! (this is for everyone –do not dismiss worries/concerns. Typically when someone loves someone, they are pretty observant and usually right.)
There is a Task Force leading this effort. Kathy Matthews, MD is Chair. Brian Denger is Project Manager. Task Force Members include representatives from Professional Societies and Advocacy organizations - MDA, PPMD, CMD, SMA.
Holly Peay is the PI on this grant. She is now part of us, part of PPMD. If you see her in the hall, please say hello.
Through DuchenneConnect and the PPMD Community site, we heard stories. Stories about delays in diagnosis…months…years at times. We thought about what we might do to help. We had some experience.
We submitted a grant to the Center for Disease Control (CDC) and were granted a cooperative agreement. The goal of the grant is to educate, to raise awareness about muscle weakness, and to impact the diagnostic odyssey. Our initial experience was in Mississippi. We developed partnerships with healthcare professionals likely to see children in the early years of life (ages birth to 5 years). We wanted to understand what happens when mom, dad, gram, or anyone really, expressed concern about a child’s muscle strength. What words did they use? What happened when they raised concern with the doctor (nurse, physician’s assistant, healthcare professional, teacher).
And we learned that some of the time, concerns are dismissed. We learned that some doctors/healthcare professionals are not exactly sure what they are to look for. We learned that some of the time, referrals to specialists are difficult with long waiting times. We learned that sometimes the response is to send the child to a PT or put them into an exercise program to improve strength. And the diagnostic odyssey continues. Typically mom, dad, gram’s worry is relieved for only a short time because they see something is still not quite right.
With this current cooperative agreement, we are expanding our horizons. Our goal is to educate healthcare professionals and this time, not in just a specific state but rather now through the professional societies. There are too many acronyms to list – but professional societies of every healthcare professional group you can think about – pediatrics, family doctors, physician’s assistants, nurse practitioners, nurses, physical therapy…you get the gist.
We have expanded this effort to include ‘primary muscle disorders of childhood’ – because the message will have greater impact. And Duchenne is the most common
We have developed common messages:
• Watch them walk, run, climb (in the confines of an exam room, sometimes it is difficult to really see what mom, dad, gram is talking about)
• Developmental Delay, include a CK (you already know this one)
• YOU CAN ALWAYS DO SOMETHING! (this is for everyone –do not dismiss worries/concerns. Typically when someone loves someone, they are pretty observant and usually right.)
There is a Task Force leading this effort. Kathy Matthews, MD is Chair. Brian Denger is Project Manager. Task Force Members include representatives from Professional Societies and Advocacy organizations - MDA, PPMD, CMD, SMA.
Holly Peay is the PI on this grant. She is now part of us, part of PPMD. If you see her in the hall, please say hello.
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