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April 2014 Blog Posts (47)

New Duchenne Therapies in Development

As we finalize the agenda for this year’s Connect Conference, I am once again amazed at how much of a challenge it is to squeeze in all of the talks on different therapeutic approaches to treating Duchenne—in fact, there are too many to cover comprehensively this year. Sometimes it’s good to remember that mutation-specific approaches like exon skipping, although promising and…

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Added by Sharon Hesterlee on April 24, 2014 at 12:30pm — 3 Comments


Staff
At Long Last, A Victory!

One Life on this earth is all that we get, whether it is enough or not enough, and the obvious conclusion would seem to be that at the very least we are fools if we do not live it as fully and bravely and beautifully as we can.…

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Added by Pat Furlong on April 21, 2014 at 6:01pm — 1 Comment


Staff
I Am Appalled

Watching my niece when she got the confirmation that her son was Duchenne was horrible, and knowing that this exquisite child will not live more than a quarter century after deteriorating for much of his young life is emotionally excruciating. Please put approval of ANY drug or treatment to help the lives of these unfortunate children on the fast track! I am appalled by the fact that my country has not put the alleviation of such suffering as top priority when we have so much…

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Added by PPMD on April 21, 2014 at 3:00pm — No Comments


Staff
Now Is the Time to Accelerate the Approval of Drugs That Can Save Our Sons



Now is the time... My son Dusty is 21. Duchenne muscular dystrophy has stolen much from him, his ability to walk, to hug his family, to move, to lift his arms, and to do the simplest of tasks, like brushing his hair. At 21, he is now faced…

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Added by PPMD on April 21, 2014 at 3:00pm — No Comments


Staff
I Am so Very Scared for the Day That I Will Have to Say Goodbye to Him



Our story is like many of the others you have received. For us, Duchenne, wasn't even on our radar. In fact, we didn't even know what it was. Wyatt at the time, was our 4th son (we had a 5th son, with the help of IVF after Wyatt was diagnosed). He was like our other boys,…

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Added by PPMD on April 21, 2014 at 3:00pm — No Comments


Staff
I Pray Every Day That the FDA Will Say Yes

My son cooper was diagnosed with dmd at 3years old, he is 5years old in July it breaks my heart every day and I cry every day. I pray every day that the FDA will say yes, it all the hope we have,being in Australia and a scientist Steve Wilton is behind making the drug, Australia dose not have the big drug companies we have to go to USA to make it happen and my son has no access too trails. So FDA USA the boys around the world have all there hope in your hands and to every FDA…

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Added by PPMD on April 21, 2014 at 3:00pm — No Comments


Staff
My BRAVE Story: I have gone from sad and confused to angry and disgruntled with the FDA

It's simple I have one grandson getting eteplirsen and doing well another, older grandson not getting the drug that could save and or improve his life. Over time I have gone from sad and confused to angry and disgruntled with the FDA and their nonchalant attitude and reaction to the needs of terminally ill people that should have the right to choose.

Grandpa Jim

Added by PPMD on April 21, 2014 at 2:37pm — No Comments


Staff
My BRAVE Story: Their lives depend on it

Our journey with Duchenne began in July 2012, just weeks before our son's 2nd birthday. Since then, our life has changed drastically and taken us in a new direction. One that we were not expecting or thought we could handle. Brendan is a true blessing. A sweet, loving, and energetic little boy! Although he is in the "grace period" of DMD, his INCURABLE and FATAL diagnosis altered the course of our family's lives forever. Devastated and eager to care for my son, I left my beloved…

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Added by PPMD on April 21, 2014 at 2:35pm — No Comments


Staff
My BRAVE Story: Please help us find hope

I grew up right in the middle of a Duchenne family. My two cousins had it at the time as well as my brother, my best friend. Early on all I knew was that my brother could not walk, little did I know that was the very least of it. Duchenne slowly and brutally made me an only child at 15. Duchenne made it so my brother could not run, skate, walk, or play with other kids. Duchenne made my brother feel lonely, depressed, and unloved. Duchenne took my brothers ability to breathe on his…

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Added by PPMD on April 21, 2014 at 2:34pm — No Comments


Staff
My BRAVE Story: I don't wanna lose my son to this disease

My first born was diagnosed with dmd,he passed away at 8yrs old in 1999,my other two sons dont have dmd,but my last son shawn does,it affects heart,lungs every muscle in the body,shawn is 8 diagnosed at 3weeks old,hes in a wheelchair,uses bipap at night,cough assist,oxygen if needed.he use to walk,talk eat my mouth,climb walk try to run,he got sick with something and took seizures for 3weeks straight in picu,he is not only battling dmd he battling global brain damage due to…

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Added by PPMD on April 21, 2014 at 2:30pm — No Comments


Staff
My BRAVE Story: We pray that this will be the first generation of DMD boys to live, instead of the last generation to die

My story began many years ago when my baby brother was diagnosed with Duchenne MD at the age of 4. I was six and we had two older sisters. My parents were shocked. They had never heard of this rare disease. The doctors didn't know what to do and offered no where to turn. They drove many hours each time to a children's hospital two states away to see a neurologist, a physical therapist and to have the surgeries done that would hopefully help. That was over 30 years ago and boy have…

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Added by PPMD on April 21, 2014 at 2:30pm — No Comments


Staff
The DMD Community Needs Hope Again

Simon was such a happy baby,he flashed his one dimple on his second day of life, while he dreamed of a life so sweet. Skinned knees, bruised shins and forehead, terrifying tumbles down the stairs, but the tears never lasted long, he was one resilient boy. At six his problems were given a name: Duchenne.…

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Added by PPMD on April 21, 2014 at 2:30pm — No Comments


Staff
My BRAVE Story: Right now HOPE is what we focus on; it is all we have

We began this journey with DMD in July 2013 when our 7-year-old son, Caeden, went to Children's hospital for a neurological evaluation and blood tests were conducted. We received a call several days later that his CPK levels were extremely elevated which indicates a muscle disorder. Caeden had genetic testing done to determine the form of muscular dystrophy and told we would get the results in about a month. We received the call about a month later which diagnosed Caeden with…

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Added by PPMD on April 21, 2014 at 2:30pm — No Comments


Staff
My BRAVE Story: This killer disease needs to be stopped

I lost my beautiful son to Duchenne, four years ago. My son was stolen from me, and the same disease that claimed his life, continues to claim so many more children's lives.DMD is 100% fatal, no doubt about it, we will continue to loose children until someone stops this killer. Approve the use of Eteplirsen, please, what if it was your boy? This drug is helping twelve boys and has had no side effects. We have nothing to loose only to gain. My son Nicholas is in heaven and I will…

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Added by PPMD on April 21, 2014 at 2:29pm — No Comments


Staff
My BRAVE Story: Three of our four sons have Duchenne Muscular Dystrophy

My name is Betty Vertin. I live in Hastings, NE with my husband and five children. We have 12 year old daughter Lexi and 4 sons; they are Max, 8, Chance, 6, Rowen, 5, and Charlie, 3. In July of 2010, when I was just 10 weeks pregnant with our youngest, our son Max was diagnosed with Duchenne. In a matter of minutes a specialist told us what no parents ever want to hear: that our son would be wheelchair bound by 9, need our help with activities of daily living as a teenager, and…

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Added by PPMD on April 21, 2014 at 2:28pm — No Comments


Staff
My BRAVE Story: Kids should not die at 16

I don't think there is enough space or time to explain how DMD has effected our family. Our son Daniel was diagnosed in 2000 when he was 6 years old. We were devastated, especially since there was no family history of the disease. He died 3 years, 6 months and 14 days ago today. That day is etched into my memory just as the day that he was born is. We had a total of 16 years with my darling boy before he passed away.

Daniel was very creative. He loved to draw, play video…

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Added by PPMD on April 21, 2014 at 2:23pm — No Comments


Staff
My BRAVE Story: It is our only hope right now to buy us time

My name is Jackie Loehlein and I have a 7 year old son with Duchenne Muscular Dystrophy. This is a fatal muscle wasting disease that to date there is no cure for this disease, but there are medications that have been proven to slow down the disease. My son has a mutation called Nonstop codon or Nonsense mutation which means he does not produce the protein dystrophin at all. The medication that is out there right now for his specific mutation is Ataluren, which has shown some…

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Added by PPMD on April 21, 2014 at 1:24pm — No Comments


Staff
My BRAVE Story: He will die because of this disease

My son Seth is almost 14 living with DMD. Everyday my heart aches watching my son loose abilities he once had. Watching him try to do things that should be easy yet for him are huge tasks that purely exhaust him. Unless you are up close to one of the children dealing with this you cannot imagine what it is like. Seth used to run and play almost the same as a healthy child then it got harder and harder and he got into a wheelchair full time by 11 years old. Myself, my husband, and…

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Added by PPMD on April 21, 2014 at 1:21pm — No Comments


Staff
My BRAVE Story: We have two sons with Duchenne Muscular Dystrophy

We have two sons with Duchenne Muscular Dystrophy, James is 13, and Jacob, who has a dual diagnosis of DMD/autism is 9. James was recommended for a trial two years ago but learned that his specific mutation did not qualify. So we must continue to wait for the next exon skipping drug to begin trial stage and expect it to be many years before it will get approved by the FDA.

I fear the progression of this muscle wasting disease in our boys' bodies during the interim will…

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Added by PPMD on April 21, 2014 at 1:20pm — No Comments


Staff
My BRAVE Story: It can happen to anyone

It can happen to anyone. Duchenne knows no borders of geography or ethnicity. My beautiful boy had trouble walking, getting off the floor. He never crawled. Doctors assured me "He'll grow out of it." My son's physical abilities peaked at 7, but he frequently fell even at a young age. His body could never keep up with his dreams and desires. It was with bittersweet relief when he broke his ankle at 10 and stopped walking. That's right. I WANTED my boy to use a wheelchair. NO MORE…

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Added by PPMD on April 21, 2014 at 1:17pm — No Comments

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