PPMD's Blog – December 2013 Archive (19)

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…

Continue

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

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…

Continue

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

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 agenda to give you a recap. (Please note that these notes may…

Continue

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

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…

Continue

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

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…

Continue

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

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…

Continue

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

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…

Continue

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

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.

Right now, Owen goes to physical and…

Continue

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

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…

Continue

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

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…

Continue

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

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…

Continue

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

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,…

Continue

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

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 used for the liver.  Connor has the genetic mutation for Duchenne with exon…

Continue

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

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…

Continue

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

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.…

Continue

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

My BRAVE Story: We are exhausted

Dears,  I am a father of fantastic young boy, 8th years old. As a child who are just entering his life he has many dreams and plans: who to be in a future, what to do in the future... However, the future may not be for him, at least this future he things about. He is DMD affected. He is already excluded from the activities that his friends do (e.g. futball, othe sports...). He feels worse than others. Decrease of his strength and increase of his awareness make him finding it as…

Continue

Added by PPMD on December 11, 2013 at 9:38am — No Comments

My BRAVE Story: Our grief and anguish are felt every single day

The news that my 3-year old grandson had Duchenne literally took my breath away.  For months, my daughter, son-in-law, and husband could barely function with this earth shattering diagnosis. 

Even today after 6years, our grief and anguish are felt every single day.  As we cling to our faith that an effective treatment will be found, a dark cloud overshadows every moment of our lives.  Imagine watching the most beautiful child in the world stand on the 3rd porch step, and…

Continue

Added by PPMD on December 11, 2013 at 9:35am — No Comments

Together We Can Make a Difference

Why the December FDA Policy Forum is important to every Duchenne family – from rare mutations to Exon 51



Guest post Lance Hester. Lance is a PPMD Board Member and lives in Gig Harbor, Washington, with his wife, Janelle, and their two boys, Brayden and Micah. Micah was diagnosed with Duchenne in 2005.…

Continue

Added by PPMD on December 9, 2013 at 1:00pm — No Comments

In Loving Gratitude

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD and mom to Hazel, age 8, and Rain, age 6. Rain has Duchenne muscular dystrophy. Follow Ivy at her blog, Living …

Continue

Added by PPMD on December 6, 2013 at 8:00am — No Comments

Monthly Archives

2018

2017

2016

2015

2014

2013

2012

2011

2010

Need help using this community site? Visit Ning's Help Page.

Members

Events

© 2019   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service