PPMD Community

October 2008 Blog Posts (18)

The Monkey on Our Backs

Yesterday, I was looking at some baby pictures of my grandson Cole, and all of a sudden this feeling came over me. I didn’t recognize what it was at first, but then I realized it was a sense of feeling free, and I felt it with every fiber in my body. It was like I was transported back in time to a few years ago, a time before this horrible diagnosis first took over our lives. I remembered what it was like to look at my grandson and just enjoy him for him without that bittersweet feeling of… Continue

Added by Terry Porcaro on October 28, 2008 at 10:26am — 2 Comments

Just need support

I have 3 sons with DMD. Jeffery chris and Jo. 16, 6 and 3. I don't know where to start. I'm a working single mom and going to school full time to try and give my kids a better life. I don't know how to balance it all, I don't have time to be deppressed or even try to think about what the future holds for my boys. How do we balance it all IEP"s PT/OT Doctors App. I feel like I'm falling apart I think about it every day how much longer do I have with them. Is today the day that one of my boys… Continue

Added by Teresa on October 27, 2008 at 9:12pm — 6 Comments


Staff
blog within a blog

This is a blog within a blog. I realize the blog should be my own words, but then again, I found Jill's words say exactly what I think. I just attended the exon skipping meeting at Banberry Center. I think we all agree that exon skipping is a promising strategy for DMD, but it isn't simple and it isn't tomorrow. Prosensa is moving forward. They are involved in a dose escalation study in 3 sites and will analyze the data. The next step is expanded sites (to include the US), this time looking at… Continue

Added by Pat Furlong on October 20, 2008 at 2:37pm — 4 Comments

Interview with Gerard Platenburg, President of Prosensa, about the Dutch exon skipping trials.

Hallo, I just joined this PPMD Community and send regards to all participants. Many of you will know me because I am writing reports on the many research approaches for a therapy of Duchenne dystrophy.



Thank you, Pat, for your so immediate messages from this very important exon-skipping conference in Cold Spring Harbor. In this context, everybody might be interested to see the interview which I recorded with Gerard Platenburg at the PPMD meeting in Philadelphia last July. Gerard… Continue

Added by Scheuerbrandt, Guenter on October 20, 2008 at 12:04pm — 1 Comment


Staff
Opportunity Knocks!

COACH TO CURE MD



Here's the thing. Long ago, my husband played football for Notre Dame. My boys, Chris and Patrick loved that golden dome. They had hopes of attending Notre Dame. They watched every game. I watched them and while they were cheering for Notre Dame, I was praying that they would be able to walk the length of the football field. Just once.



Duchenne has changed. We stand at the forefront of promising clinical trials. We have been instrumental in making this… Continue

Added by Pat Furlong on October 20, 2008 at 10:44am — 9 Comments

Frustrated and upset!

I apologize for this vent by I must as you are all aware of the healing properties of venting...



My son has been diagnosed with DMD in March 2006. Then un-diagnosed early this year. We have no diagnoses but yet he has symptoms. They want to do another muscle biopsy but I resist. I can't let my boy go through another biopsy when there are so many other non-intrusive ways in finding it. What can they see now that they didn't see in 2006? We have all the lab results studying the muscle… Continue

Added by MarcosDad on October 18, 2008 at 9:51am — 9 Comments

Looking for assistance in Florida

Hello, I am posting here to try to find some kind of help for my mom and dad. As you can see from my profile, I am facing many challenges here and need some guidance or advice. I'll give the background first. My brother is 22 and according to Florida laws and statutes, he does not qualify for any in-home assistance. He only had Medicaid and neither of my parents have health insurance so that is all he has. And as we all know, Medicaid pays for the minimum necessities needed to accommodate my… Continue

Added by Jen on October 16, 2008 at 11:08pm — 1 Comment


Staff
exon skipping continued

Hello Again,

The meeting is going well. It is clear that antisense oligonucleotides are a promising strategy in DMD. It is not simple and there will be some bumps in the road. I also realize there are a lot of questions about duplications and early exons. Steve Wilton suggested that he has developed more than 650 chemistries taregeted agains a range of exons from 2-78, but that there are massive variations in efficiency. Keep in mind, this approach is expected to slow things down,changing… Continue

Added by Pat Furlong on October 16, 2008 at 7:59am — 7 Comments


Staff
Antisense Meeting at Banberry Center

I am participating in the exon skipping (aon) meeting at Banberry Center. All of the leaders in the AON field are here as well as the basic scientists invovled in this space. We are here to discuss issues related to antisense as a potential therapy for dmd. You are already aware, prosensa is currently involved in a dose escalation trial in 3 locations in Europe. The trial involves 1518 patients, skipping exon 51. It is dose escalation trial and currently recruiting patients. This trial is to… Continue

Added by Pat Furlong on October 15, 2008 at 4:06pm — 2 Comments


Staff
What't the deal on Losartin

You are all aware of the Losartin paper by R. Cohn and colleague and like many other drugs/supplements/nutraceuticals, we continues to discuss this within the community and ask various physicians to prescribe Losartin for our boys. And some parents are giving it to their son, with or without the blessing of the physician. So, the botton line is that there is no evidence other than on study on the mdx mouse. And you have already heard the words, "there is a great deal of difference between the… Continue

Added by Pat Furlong on October 13, 2008 at 10:22pm — 26 Comments

With a little help from my friends.

I dropped Matthew off at his Homecoming dance this weekend. Whoever coined the phrase "mixed emotions" probably didn't realize how true it is for me in situations like this. After unloading Matthew from our van and leading him through the line of students to get to the accessible door I stood and looked at all the young people crowded inside. I shuddered wondering how would they all get out in an emergency, but also knew Matthew would be with great friends and have a fun… Continue

Added by Brian Denger on October 12, 2008 at 8:58pm — No Comments

First MDA Clinic Visit

Ryan had his first MDA clinic visit at Duke University Medical Center in Durham, North Carolina on Firday, October 3, 2008. We left St. Augustine at about 3:45 on Thursday afternoon. After a painfully long drive, we arrived at our hotel at about 1 am. The next day, Ryan saw the neurologist at about 8 am, the pulmonologist at 12:30 pm, and the cardiologist/geneticist at 2 pm. Over all, we left Duke at about 4:30 pm. An incredibly LONG day!!! All of Ryan's visits went very well. His lungs and… Continue

Added by Jennifer Collier on October 8, 2008 at 8:40am — 2 Comments

Coach for a Cure

Today I feel like we made a contribution for DMD. We participated in the Coach for the Cure. The Coach for a Cure campaign is an arrangement with the American Coaching League. An individual by the name of Brad Todd, has a nephew who has DMD, helped orchestrate the event. Brad has been working for two years to get PPMD on the American Coaching League's dance card if you will. This year, he was successful and today we participated in a commercial that will be aired on Oct 25th during college… Continue

Added by Char Burke on October 7, 2008 at 12:22am — No Comments

Fall is here!

So Tim has been out of the hospital now for about a week and few days and so far he is still having difficulty swallowing, coughing and occassionally breathing. But he has improved big time. now to get him back into classes at UF. The thing is he still so exhausted and drained that he does not have the energy to studdy or do his school work. I'm not sure how else to motivate him except that he is on academic/financial aid probation so he has to do well. I see that as to much pressure for him… Continue

Added by Dee on October 4, 2008 at 7:07pm — No Comments

T-Ball

Last night was an exciting night for Sam. He's just started playing T-Ball this fall. My husband is the coach. At last night's game, Sam scored 3 runs!!! I missed the first two because Abigail and I were at karate, but I made it there in time to see the third. It truly brought tears to my eyes. He couldn't have been more pleased with himself.



Here's a few pictures of my little T-Ball players.…

Continue

Added by Laurie Paschal on October 4, 2008 at 10:02am — 1 Comment


Staff
Partners, family members, friends - all respond in different ways at different times. I had to figure a way to understand.

Different views of Duchenne. Duchenne walks in the door and everything changes. Everyone’s heart is breaking in different ways and at different times and everyone has an opinion. Fingers point. Like sleuths we want to find out what happened, how it happened and where this diagnosis came from.



Moms wear the guilt no matter. Moms feel guilty as if they missed something along the way, some bit of information that might have prevented the diagnosis. They roll up their sleeves. They will… Continue

Added by Pat Furlong on October 2, 2008 at 8:07pm — 7 Comments

I have joined this site for a special reason. Our friends the "Mannings" have found out that their son Cragie, has DMD. My husband and Cragie's father work together, and over time we have all become …

I have joined this site for a special reason. Our friends the "Mannings" have found out that their son Cragie, has DMD. My husband and Cragie's father work together, and over time we have all become close friends.

So sad...the Manning's have found out that their youngest son Marc, has DMD also. Cragie and Marc, are so sweet, they are always so happy to see you, they make my heart melt. I love those little guys.



For all you dealing with this...I pray for you all, and your sons,… Continue

Added by Joniann Bencivenga on October 2, 2008 at 2:03pm — 2 Comments


Staff
Impact of Re-authorization

I find it important to sometimes take a look back. The MD CARE Act of 2001 was passed and signed into law in record time.. 11months. It was introduced in February, 2001 and signed Dec. 2001. This legislation put MD on the radar screen of the NIH, Dept. of Defense and CDC. But more than that, it caused a philosophical shift in attitudes, ideas and possibilities. It stimulated reseach and industry interest and insisted on data collection and the development of Standards of Care (Care… Continue

Added by Pat Furlong on October 1, 2008 at 4:26pm — 2 Comments

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