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December 2013 Blog Posts (27)

Ups and downs..

Sometimes it feels as though nothing is wrong, at all. And that's what's the most baffling - my son is racing around, playing with friends, running like the clappers, full of energy.. you'd just never think he had a muscle disorder going on in the background. And then he flops on the couch and looks absolutely knackered, and there it is - the little reminders. I guess it'll be this way for a while. But who knows? 2014 really requires a new mindset: one day at a time. Easy to write it here..…

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Added by Frankie Butler on December 30, 2013 at 6:21pm — No Comments

Razor E100

Thanks to all y'all on here, I bought Dash a Razor electric scooter for Christmas this year - it's awesome! I might have to get a grown-up one for myself

Added by Frankie Butler on December 24, 2013 at 9:24am — No Comments


Staff
Halo HT-100 Update

We are heartbroken to learn more discouraging news about another Duchenne therapy today, HT-100 from Halo Therapeutics. This community continues to face tremendous disappointment.



As part of Halo’s efforts to accelerate HT-100 development, Halo was conducting a toxicology study in dogs in parallel with the clinical program. In this study, the dogs got higher exposures (levels of…

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Added by PPMD on December 23, 2013 at 2:38pm — 1 Comment


Staff
Current Status of Drisapersen Studies

Analyses and assessment of next steps are ongoing

 

PPMD just received the following update from GSK regarding the current status of their drisapersen study:

Dear Patient Group Representative,



On the 20th of September this year, we shared the results about our Phase III study (DMD114044) of drisapersen in boys with Duchenne Muscular Dystrophy – we did not see a statistically significant difference in 6 Minute…

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Added by PPMD on December 19, 2013 at 10:30am — No Comments

Exon 30

We now have a diagnosis: Dash has an in-frame deletion of exon 30 and this is "consistent with Becker diagnosis".

This is relative good news for us and him, I suppose - not great, but not Duchenne. It's interesting, because although this means he is luckier than some, I was right there and ready to be told that it was Duchenne - and that feeling hasn't really GONE anywhere. I still feel furious at the injustice of it all, desperate to do something. Whether my own child is…

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Added by Frankie Butler on December 18, 2013 at 1:41am — 1 Comment


Staff
Featured Voices of 2013

Each month for the last couple of years we have featured people with Duchenne who have made an impact on their community and our community in some significant way. Whether working with their parents to host a fundraising event, participating in a race, speaking in Washington, D.C., or sharing their story…

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Added by Will Nolan on December 16, 2013 at 2:30pm — No Comments


Staff
Duchenne Policy Forum Recap

At yesterday’s Duchenne Policy Forum, Vice President of Research Sharon Hesterlee, PhD, tweeted throughout the day, reporting to the community what transpired during this historic meeting. We have compiled these tweets and fit them into the…

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Added by PPMD on December 13, 2013 at 2:30pm — 1 Comment


Staff
Yesterday's Duchenne Policy Forum

Silver Springs, Maryland

12/12/13  8:30 AM 

There are days when the stars align just right. Seems to me, December 12, 2013 was one of those days.

Eighteen members of the FDA arrived and were seated around a U-shaped table near the front of the…

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Added by Pat Furlong on December 13, 2013 at 12:30pm — 1 Comment


Staff
My BRAVE Story: Every day our hearts are beaten and bruised knowing what DMD will do to our sweet, happy child

I'd like to share my post from 7 months ago from our Facebook page - https://facebook.com/Duchenneroadmap

June 8, 2013

Right now on this quiet, easy Saturday morning, I'm listening to Noah play with his toys on his train table. He sounds like any other 4 year old boy, making up an imaginative story about constructing a new airport and acting it out with the various vehicles. I can almost forget... Almost forget that ten…

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Added by PPMD on December 11, 2013 at 11:45am — No Comments


Staff
My BRAVE Story: I have Duchenne Muscular Dystrophy

Deletions 46  52  Need 53 or 22  46  I live across the pond in the United Kingdom in a City named Manchester I live at home with my mother. My father passed away in March 2012. I have Duchenne Muscular Dystrophy I'm 26 years old. I'm currently unemployed however I spend most of my days fundraising and raising awareness of Duchenne.  Even though the current trial won't directly affect me the next one will impact my life completely. More delays to the current trial mean waiting even…

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Added by PPMD on December 11, 2013 at 11:30am — No Comments


Staff
My BRAVE Story: You have the power to help us

Our 6 year old son, Tom, was diagnosed with Duchenne Muscular Dystrophy in December 2011 and our lives, and that of our wider family, have never been the same.  There is now an ever present weight of sorrow carried by us his parents as well as grandparents, godparents, uncles, aunts, cousins and step-brothers..  Although our son is still mobile and despite daily physiotherapy, night splints and hydrotherapy, we cannot stop the onslaught of his ever tightening calf muscles which…

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Added by PPMD on December 11, 2013 at 11:30am — No Comments


Staff
My BRAVE Story: Please FDA do not take away my hope

I remember very clearly the day we received the devastating news are beloved Grandson had Duchenne Muscular Dystrophy. Now I knew of Muscular Dystrophy, I had watched may telethons over Labor Day weekends and of course seen firefighters collecting for it over the years. But this Duchenne what was this? It did not take long for me to discover how our lives would never be the same again. But I left the hospital full of hope. He was young a cure would surely be found in todays…

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Added by PPMD on December 11, 2013 at 11:00am — No Comments


Staff
My BRAVE Story: We are scared and sad for what the future will hold

My son, Owen, was diagnosed in July of 2012, 2 weeks prior to his third birthday.  We noticed difficulty in climbing stairs and running but thought he just had flat feet.  We had never heard of DMD and on 7/25/12 our lives changed forever.  Since the diagnosis, we have been on an emotional roller coaster.…

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Added by PPMD on December 11, 2013 at 11:00am — No Comments


Staff
My BRAVE Story: We hope that he is lucky enough not to experience the full effects of this life-robbing disease

Our son Eliot was diagnosed with DMD in May 2013. He turned two in November. Although we are at the start of a long and difficult journey, we are well aware of the clinical research that is being undertaken around the world to combat this disease. We hope that something similar to the Lung Cancer Master Protocol for Clinical Trials can be established for Duchenne, in order to speed delivery of treatments to all boys. Without this coordination alongside accelerated approvals we…

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Added by PPMD on December 11, 2013 at 11:00am — No Comments


Staff
My BRAVE Story: It may be your child that is fighting for their life someday

I have this beautiful grandson. His name is Lucas Webster. He is 13 years old. 10 years ago our lives came tumbling down, when Lucas was diagnosed with DMD. God gave him the mind and spirit to be a outdoor everything. He has always loved fishing, hunting,raising gardens and flowers. He ask me one day," MaMaw when I get my medicine that will take this disease away, can I run and run and run fast," and I said, YES you will. But he is loosing his spirit. And his body is failing…

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Added by PPMD on December 11, 2013 at 10:53am — No Comments


Staff
My BRAVE Story: Urgent message to the FDA

Dear PPMD and FDA,

Like many parents have, I'll start by giving my son's age. Miles is 6 years old and in 1st grade. The significance of age is obvious to all of us. It is a reliable marker of where he is in a journey with a tragic arc, where every day, month and year counts.

Miles is a happy kid. At 6, he is able to walk and play, participate in most activities at school and at home, dress himself, travel, ride the bus and swim in the ocean. That's not to say he doesn't have…

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Added by PPMD on December 11, 2013 at 10:17am — No Comments


Staff
My BRAVE Story: My son is brave

My sweet 5 year old completed the US drisapersen trial a few months ago.  He has some mild cognitive delay, and I don't think he ever really understood why he had to drive 3 hours every week to get a blood draw and painful injection.  However, he managed to maintain a cheerful attitude throughout the trial (except for the general anesthesia and myriad pharmacokinetic blood draws for his 2 muscle biopsies).      We now know he was receiving the low dose of the investigational drug,…

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Added by PPMD on December 11, 2013 at 10:00am — No Comments


Staff
My BRAVE Story: If it is working for even a small fraction of our boys, then let the drug be available

My son, Connor, has Duchenne Muscular Dystrophy and he is 8 year's old. We've known since he was 2, only because one blood test showed elevated liver enzyme levels but it wasn't anything wrong with his liver. His muscle break down elevates the same tests that they basic blood work tests…

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Added by PPMD on December 11, 2013 at 10:00am — No Comments


Staff
My BRAVE Story: It's hard to watch him struggle

My beautiful son, Hunter, was born on December 6, 2005. He had the biggest blue eyes and still does, whenever he lays those baby blues on you, I cannot help but hope and hope that somehow someway a miracle is going to happen.   Maybe our miracle has already come and we are only allotted one per lifetime, but I do not believe his has happened yet. Unfortunately, Hunter was diagnosed at 21 months with Pre B Lymphoblastic Leukemia, on September 17, 2007, he was flown by helicopter by…

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Added by PPMD on December 11, 2013 at 9:51am — No Comments


Staff
My BRAVE Story: How long does it have to take?

Imagine having a healthy child. You watch them develop slow. You take your child to doctor or several doctors and have test after test. The tests come back your child dx with duchenne md. You carry on trying to put the fear of one day losing your child to this disease locked in the back of your mind.(you love this child while you can) As years go on you will watch this child try to run when they can only walk fast. One day they can no longer get up off the floor by their selves.…

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Added by PPMD on December 11, 2013 at 9:41am — No Comments

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