PPMD Community

November 2009 Blog Posts (27)

Looking For Alternatives....

Hello everyone hope all is well...I am looking for any information from anyone who can give some. My son just turned 8 and was dx with Duchenes last November...For the past six months I have been trying to get in with Dr. Wong unfortunately 4 weeks ago I was laid off and now have filed for medicaid because our cobra policy is 1500 a month and i am the only one workiing in our 6 person household. Does anyone know from an insurance or financial perspective how Dr. Wong works? Does she take… Continue

Added by Phyllis on November 28, 2009 at 3:51pm — 4 Comments

Just Joined

I only recently joined this community. My name is Andrew, and my wife Mandy and I have two sons, Hayden who just turned four and Cameron who just turned one. It was early this year, when we learned our nephew, Noah, had been diagnosed with a form for muscular dystrophy. To be honest, at the time, I knew nothing about it. I asked my wife how serious it was and could tell right away it was very serious. It wasn't long after that we learned it was Duchenne, and from there I was able to look it up… Continue

Added by Andrew Kerr on November 26, 2009 at 4:24pm — 4 Comments

Thanks

thank you Pat, for staying with us and for leading us. Happy Thanksgiving!

Martin & Melissa Daniel

Added by Martin Daniel on November 26, 2009 at 10:36am — No Comments

Thanks

Thanks to you for giving us hope. I am a grandmother of a beautiful little boy recently diagnosed with duchenne.. People like you inspire me to keep up the hope and do as much fundraising and political action as possible to raise money for research and keep those in Washington DC. aware of the need for pushing funds for duchenne md. and to NIH for research. I give thanks to those scientists worldwide working on treatments and cures. Most of all I give thanks to all our boys who are the most… Continue

Added by Harriet Meermann on November 25, 2009 at 5:42pm — No Comments

Staff A moment for thanks

We spend most of our lives in running shoes. This past year in particular, there were added worries, and it felt like we had to run stronger and faster, often without taking a moment to catch our breath.

On Thanksgiving, it is important to loosen those laces, put the shoes in a corner, and take a moment to bow our heads and give thanks.

I am thankful…

•…for amazing advances in research. Our End Duchenne Grant Award Program invested almost $1 million in research,… Continue

Added by Pat Furlong on November 25, 2009 at 10:11am — 19 Comments

Staff HOPE – finding the Therapeutic Dose

It’s not easy. The word Duchenne enters our lives. We search the internet, seek out expertise, connect with researchers, clinicians, industry, other families, set up our internet tools (Google alerts, RSS feeds, list-serves, pubmed search criteria, industry alerts, etc) and live each day hoping to see our ‘in-box’ overflowing with news, with HOPE.

On any given day, you reassure yourself that your son is young, may have a certain mutation, may have a certain degree of muscle function,… Continue

Added by Pat Furlong on November 23, 2009 at 3:10pm — 7 Comments

PLEASE DONATE NOW!

HLEP US BY DONATING ANYTHING YOU CAN TO THIS WEBSITE!!!!!!!!!!

EVERY BIT HELPS!!!!!!!!!!!!!!!!

click below to donate

https://www.joinmda.org/salisburylockup09/pthompso

any donations will go towards the expenses listed below

$30 - Flu Shot

$82 -1 Minute of Research

$100 - Support Group Session

$800 - Child to MDA Summer Camp

$2,000 - Assist with Wheelchair… Continue

Added by Morgan Brown on November 19, 2009 at 12:12pm — No Comments

Staff Notes from Treat-NMD Conference

Phase I/IIa study on Antisense Compound PRO051 in patients with Duchenne (van Deutekom)

Eight years in the drug development of AON. PRO051 is a 2’o-Me-PS RNA antisense oligonucleotide introducing exon 51 skipping. It demonstrated proof of concept with up to 95% dystrophin fibers in treated muscle area in Duchenne patients. 6 month safety data on PRO051 was available in monkeys and longer term studies in progress. Extensive safety data available for this class of compounds. 12… Continue

Added by Pat Furlong on November 18, 2009 at 1:34pm — 10 Comments

Staff News from Brussels

Currently in Brussels for the Treat NMD Advisory Committee for Therapeutics Conference.

How many times have we all received Google Alerts suggesting a certain drug "is potentially promising," “improves function in the mdx mouse,” or ‘halts degeneration in the mdx”? And then, silence.

And how many of us have discussed the need for a certain drug to go to trial or how many parents/families have tried to persuade or were successful at persuading their physician to write the… Continue

Added by Pat Furlong on November 17, 2009 at 4:28pm — 6 Comments

Skeptical too

Pat, I share your views on this. We certainly hope this is for real, but the details seem sketchy.

He doesn't know where the biopsy was taken from? Who is this Dr. Riordan fellow? Where did he come from?

Matthew's Dad.

Added by Martin Daniel on November 12, 2009 at 9:01pm — 1 Comment

Please Help

HLEP US BY DONATING ANYTHING YOU CAN TO THIS WEBSITE!!!!!!!!!!

EVERY BIT HELPS!!!!!!!!!!!!!!!!

https://www.joinmda.org/salisburylockup09/pthompso

any donations will go towards the expenses listed below

$30 - Flu Shot

$82 -1 Minute of Research

$100 - Support Group Session

$800 - Child to MDA Summer Camp

$2,000 - Assist with Wheelchair or Leg… Continue

Added by Morgan Brown on November 12, 2009 at 12:21pm — No Comments

Please Help

Added by Morgan Brown on November 12, 2009 at 12:21pm — No Comments

Staff "MD Breakthrough" Dr Riordan - update

I spoke with Dr. Riordan yesterday. I found him very open and willing to discuss his work.

To date he has performed the cell transplant procedure on three young adults with Duchenne. Ryan is 23. Another young man was 28. The procedure was considiered Compassionate use.

Dr. Riordan is using MSCs (human mesenchymal stem cells). The origin of these cells is from bone marrow donors, age 18-65 and/or umbilical cord cells. Dr. Riordan said there is greater potency of cells from… Continue

Added by Pat Furlong on November 12, 2009 at 7:09am — 2 Comments

Staff "MD Breakthrough" Dr Riordan - update

Added by Pat Furlong on November 12, 2009 at 7:09am — 12 Comments

Staff MD Breakthrough - What to Make of It

Whenever I hear the words "MD Breakthrough', my heart skips a beat. Is it really? Often, it is in reference to a study in the mdx mouse. We can cure mice. But people are not mice... just ask Victor Dubowitz. And when the 'breakthrough' talks about a young man with Duchenne, it feels like we are all standing at Attention!

Over the last couple of days, many of you have seen a news story that recently aired about a young man named Ryan. I wanted to share with you my thoughts… Continue

Added by Pat Furlong on November 10, 2009 at 4:28pm — 47 Comments

Stop putting Things on Hold...& Just do it!!

IF you are new here to the PPMD community, or even if you are not new to the community, you might noticed the set of photos that we as parents share of our boys.

Look at all those faces. They are smiling. They are innocent. These are the faces of our boys who has their mom and dads and grandparents, uncles and aunts, siblings and cousins that fight to end Duchene. They fight for their lives, because of a disease that robs them the ability to do tihngs for themselves. While many stay… Continue

Added by Dee on November 10, 2009 at 6:30am — 5 Comments

December South Texas FACES Meeting

All,
Our December South Texas FACES meeting has been scheduled for Dec 5th. Please check out the evite and let us know if you can attend.

http://www.evite.com/pages/invite/viewInvite.jsp?event=OCDCFMSEVDWBOSMHGMLO&inviteId=UZGGZSQRMKAKQZETPHZP&showPreview=false&x=309580352

Laurie

Added by Laurie Paschal on November 9, 2009 at 5:22pm — No Comments

Staff Joshua Arndt

Joshua died last Monday. There are no words to soften the blow. Losing one of our sons hurts all of us. The world is just not the same, never will be. We can ask ‘why’ and say all of the expected things, but it does not sync. Losing Joshua is unacceptable.

Our heart is broken again. He was one of us, he is our son. His parents are dear friends, colleagues, fighters in this difficult battle to EndDuchenne.

Our sons teach us everyday of their lives. They teach us how to… Continue

Added by Pat Furlong on November 9, 2009 at 10:30am — 7 Comments

Finding the words

I haven't posted a blog in a while. I'm never sure what to say. My words are never as eloquent as Pat Furlong's or Brian Denger's. The gift for words has always seemed to escape me. My gift for finding the right words was really put to the test this week.

On Tuesday morning, on my way to my office in Buffalo, I found out that one of the families that helped me fundraise for Coach to Cure MD in upstate NY had their 4 year old son pass away without warning on Monday. Devastating news!… Continue

Added by Christine Piacentino on November 8, 2009 at 11:36am — 4 Comments

Participation

I have a picture of Matthew and Patrick at my desk at work taken about three years ago when Patrick was still able to walk. Patrick is standing and leaning against Matthew who is sitting in his power chair. Patrick's arm is around Matthew's shoulder and they are both smiling. The look on Patrick's face is self assured, almost protective, and Matthew looks relaxed and happy next to his brother. The photo reminds me of how much my sons help each other and depend on each other for… Continue

Added by Brian Denger on November 8, 2009 at 5:55am — 1 Comment