PPMD Community

December 2009 Blog Posts (17)

Staff Happy New Year

Dear Friends,

It is the last day of 2009, the end of the decade. It seemed every conversation started with 'in this economy' and we all worried what those words might mean for progress in Duchenne, for buying time. As a community, we held tight to each other, to our sons and to our hope. We end on a high point:

Ataluren pivotal trial

Validated outcome measures for ambulatory boys

Antisense oligonucleotides in systemic trial (Prosensa and Avi)

Alaluren trial for… Continue

Added by Pat Furlong on December 31, 2009 at 3:29pm — 5 Comments

Merry Christmas!!!

Merry Christmas from the Paschal family....

Added by Laurie Paschal on December 23, 2009 at 9:13pm — 1 Comment

Looking for some pictures to put a face to the cause

Hello All,

Some of you may know us and some may not. My wife and I founded a small nonprofit, Ryan's Quest after learning of our son Ryan's diagnosis of DMD 2 years ago. Like many of you we threw ourselves into finding ways to raise money and awareness for DMD. We have a great relationship with PPMD and have helped fund a handful of projects and will continue to do so in the future. This February we are hosting a Valentine Ball to mark our 2 year anniversary of founding Ryan's… Continue

Added by Dave Schultz on December 21, 2009 at 2:11pm — 4 Comments

December 2009 Update

Last week, we made our bi-annual trip to Cincinnati for Bradley's DMD appointments. Overall we got a good report. They are amazed that he is maintaining strength and that he is still getting around so well. It did take him a little longer to get up from a sitting position on the floor; this does concern me some but he did his 30 feet slightly faster this time so that was a plus.

Getting up from sitting on the floor:

June 2008-4.8 seconds

January 2009-4.79… Continue

Added by Lisa Jones on December 17, 2009 at 10:00am — No Comments

Staff The "C" - Part 1

My friend Mela says I signed up for this a long time ago, several lifetimes perhaps, in a book I don’t recall and in a place I don’t remember. She believes in reincarnation, that we come back to learn things, important things, necessary things. If so, I would be interested in making the argument that we should at least get to remember what we learned in previous lives. It is only fair. I cannot argue for or against reincarnation. I simply don’t know. I can see the utility in recycling souls,… Continue

Added by Pat Furlong on December 16, 2009 at 2:53pm — 15 Comments

Best wishes to all

Continue

Added by BHARAT CH. TAMULY on December 15, 2009 at 10:51am — No Comments

Staff Welcome news!

Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

I realize it is so difficult to see trials targeting specific mutations and feel like you are on the outside looking in, wondering when it will be your son’s turn. Prosensa and AVI’s trials are moving forward as well. More trials are in the planning stages – Sildenafil, Losartin, IGF-1 and others. Still some of our boys do not fit the criteria, either… Continue

Added by Pat Furlong on December 11, 2009 at 2:59pm — 6 Comments

Christmas in the hospital

On Thursday, December 17th, our 23-year-old son Nick will be entering the hospital to have a tracheotomy. We had hoped we would never have to do this but Nick is one of the minorities that cannot use NIV. We are very concerned and wish that he wasn’t going to have to spend Christmas in the hospital but we also realize that this is in his best interest and will improve his quality of life.

When faced with something like this it is easy to get overwhelmed and let living with this… Continue

Added by Debbie Fish on December 11, 2009 at 12:12pm — 5 Comments

Staff Congratulations to Annemieke Aartsma-Rus

You may have met Annemieke at the PPMD conference. She is wonderful and brilliant and committed to our sons.

NWO has awarded Vidi-grants to outstanding research and researchers: Annemieke Aartsma-Rus of Leiden LUMC The Netherlands wins the prize!

The Dutch Organization for Scientific Research (NWO) has awarded 89 young, innovative scientists a so-called Vidi-grant. Each researcher will receive a grant amounting up to 800,000 euro aimed at developing an own line of… Continue

Added by Pat Furlong on December 10, 2009 at 9:11am — 5 Comments

My Boys Bill 41 and Chris 36

Over the next few weeks I will post as a blog a history as best as I can remember about my two boys who have DMD. Billy was born 3/15/68 and Chris was born 12/28/73. We found out that they had muscular dystrophy when billy was 5 1/2 old, just after christopher was born.

Added by Bill Watts on December 9, 2009 at 4:31pm — 1 Comment

Cincinnati.....again

We just got back from another visit to CCHMC. We learn so much every time that we go. Wyatt had a rough day the first day. He was pretty much asleep all day (because of sedation), so when it was time for us to sleep, he was wide awake. He had an MRI and lots of blood work done. He's doing so well. All the doctors, nurses and staff members we talked to were so glad to see that he is finally growing. All his tests came back normal. We are waiting on some additional blood work (not related to DMD)… Continue

Added by Wyatt's Mommy, Melissa on December 8, 2009 at 11:31pm — 1 Comment

vote for PPMD on the Chase Community Giving

Don't forget to go there and cast your votes. There are less than 1800 votes and I know there are a lot of you that may not know your vote is needed. I found this site through facebook, it is Chase Community Giving. They have 5 million dollars they will donate to the charities with the most votes. The link is www.facebook.com/ChaseCommnityGiving.

Added by Eunice Rivera on December 8, 2009 at 11:11pm — 1 Comment

Staff Care Considerations Available

Care Considerations – to be published in Lancet – is available now, online . These Care Considerations set the stage for all boys everywhere to receive optimal care. Families now have access to this document as well as an abbreviated, family-friendly version currently in development. This is your guide and your doctor's guide to good care. Your questions, your concerns are… Continue

Added by Pat Furlong on December 7, 2009 at 12:34pm — 6 Comments

Staff Notes from UPPMD Endocrine Meeting: Florence, Italy - October 28 & 29

Care and treatment in Duchenne muscular dystrophy is evolving with improved treatments and promising therapies on the horizon. Parents and young men need to be proactive about care, to understand what to do, what can be done and the appropriate time for intervention. Risk/benefit analysis must be thoroughly and carefully evaluated. It is our responsibility as a community to act as advocates for our sons in order to improve current therapies, to address gaps in care, and to accelerate the… Continue

Added by Pat Furlong on December 7, 2009 at 12:02pm — 1 Comment

Staff Josh Winheld - In memory

Saywatanayo (Shawshank Redemption)

To my friend Josh,

I’m sure heaven is a brighter place today, but our world feels pretty empty. We did not have time to say goodbye. Or time to tell you all of the things that come to mind today or how thankful we were to know you, to have you in our lives, to learn from you, to see you smile.

You had learned all of the lessons life had to teach. You shared your knowledge, spread your joy and extended your hand to all of us.… Continue

Added by Pat Furlong on December 6, 2009 at 6:54pm — 7 Comments

steroids, lisinopril and illness...

max is 7 and has been up vomiting every 30 min all night ( nothing left in his belly, but he is still heaving) does anyone know what we are supposed to do about his am meds? he takes deflazacort and lisinopril, not supposed to miss either. we have not encountered this yet believe it or not, with either boy, and i know local docs will have no idea, my boys are the only dmd kids around. thanks, jenn

Added by jenn on December 6, 2009 at 7:26am — 6 Comments

how to buy a van..

advice would be greatly appreciated...as with any aspect of duchenne, we were not truely prepared for this next step of austin(age 10) using a full power chair. he actually can walk some, but the scooter he has used for yeras was not supporting him, and we wanted him to be more comfortable as he really doesnt walk much at school, so, the chair is here, the issue is, we have no way to transport it. we had thought this would be easy, go to local dealership ( my husband happens to be friends with… Continue

Added by jenn on December 3, 2009 at 8:47am — 12 Comments