PPMD's Blog – November 2013 Archive (52)

My BRAVE Story: I think about a cure being found everyday

Our youngest son, Matthew, was diagnosed with DMD at age 2.5, we had never heard of DMD before & after researching it, we were devastated. It is hard to watch the child you love lose the ability to do things he could do last year, last month, yesterday. He is 12.5 now & continues to get weaker. It breaks my heart to see him struggle to hold his iPad or the TV remote, items we can lift & use without even thinking about it.

I think about a cure being found…

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Added by PPMD on November 20, 2013 at 11:07am — No Comments

My BRAVE Story: Risks are acceptable

In some cases, risks are acceptable to parents and to the patient

Added by PPMD on November 20, 2013 at 11:03am — No Comments

My BRAVE Story: The FDA should allow these treatments since we know the outcome of this illness

My son turns 21 this year and I want to do everything possible to keep his strength and in good health. There are a number of drugs currently being used of other illness (cancer) and I believe and hope the FDA should allow these treatments since we know the outcome of this illness. Please allow off label use of these drugs.

Added by PPMD on November 20, 2013 at 10:46am — No Comments

My BRAVE Story: Our lives have changed dramatically and our family will never be the same

My son, Owen, was diagnosed with DMD in July of 2012 - 3 weeks from his 3rd birthday.

Owen is a fraternal twin with his sister Grace and has an older brother who is 6. We thought Owen had flat feet and started with a orthopedic doctor, who told us she thought it was neurological. Owen had trouble running and going up stairs. He walked late but so did his twin sister. We saw a neurologist, had CK test done and in 2 days we received the devastating news of DMD.

Our…

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Added by PPMD on November 20, 2013 at 9:46am — No Comments

My BRAVE Story: Save the lives of our children

Hi my name is Juan Banuelos

My son is 7 years old and was diagnosed with Duchenne on 8-20-12 he has a mutation in exon 31 and it seems like he is not in the hot spot of mutated exons. And drug companies are only looking at the most common and are putting them in clinical trials which has had a lot of success and I feel hopeless in that sense the technology is out there so please put the rare exons also. On 8-20-12 I felt like I was gutted and empty my whole life change in a…

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Added by PPMD on November 20, 2013 at 9:30am — No Comments

My BRAVE Story: There Is Still Hope

Our Life With A Rare Disease Called Duchenne -  Submitted by Christine Piacentino My life with Duchenne Muscular Dystrophy started on December 31, 1997.

Today my son Jonathan is 19. On the day of his diagnosis, he was 4 years old. The doctor explained the prognosis. My son could lose the ability to walk by the age of 12, the ability to breath could be lost by 15 and my son might pass away by his mid 20s. The doctor next said that there is still hope. There could be…

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Added by PPMD on November 19, 2013 at 3:47pm — No Comments

My BRAVE Story: Relax the Requirements for Trials

My son, Kyle, is 17 years old. He has not walked since he was 12 and does not remember ever walking. Kyle is an honors student in high school, marches (in a wheelchair pushed by another student) in marching band, plays in the top band at his school, was selected by our Mayor as one of the top 100 teens in the City, and plans to attend Texas A&M University. He wants to major in engineering, as he has a very analytical mind.

Kyle is a blessing and inspiration to all who…

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Added by PPMD on November 19, 2013 at 3:29pm — No Comments

Facing Disappointment from FDA's Eteplirsen Response

By now many of you may have heard the news that the FDA has called the new drug application for the exon 51 skipping drug eteplirsen premature.  They have requested instead that a larger phase III study be conducted before they will consider the application for approval.  PPMD, which provided travel support to families who…

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Added by PPMD on November 12, 2013 at 2:00pm — 1 Comment

My BRAVE Story: The concept of risk vs. reward has changed for me

My son, Michael is 17 years old. He was 5 when he was diagnosed with Duchenne. He is our only child.



The day he was diagnosed was by far the worst day of our lives. A diagnosis out of the blue. You walk into the doctor office with a healthy happy 5 year old and you walk out with a child sentenced to death. A life filled with the disappointments of losing the ability to do one thing after the other.



It's so hard to describe the toll Duchenne takes on a life,…

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Added by PPMD on November 5, 2013 at 4:02pm — No Comments

My BRAVE Story: I am one parent willing to take an educated risk

I want the FDA to know that my son is a beautiful, seemingly healthy boy that is dying inside. His muscles aren't and can't grow until they allow us medicinces that can slow the progression of Duchenne. 

I understand the need for caution and care, but I also know that while drug companies are allowed to give medicines in other countries, our children are dying. We should be able to use studies and data from other countries without having to jump through duplicative hoops in…

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Added by PPMD on November 5, 2013 at 2:00pm — No Comments

My BRAVE Story: Our son was diagnosed just 5 years ago

Dear FDA, 



Our son was diagnosed just 5 years ago at age 9. 



We consider our family to be lucky because in the past 5 years Duchenne has only taken away our son's walking, standing, dressing and bathroom independence. So far we have managed these things.  



We are also lucky because in the past 5 years we've only had to purchase (out of pocket), one manual chair for travel, one power chair, one ceiling mounted lift, one freestanding lift for…

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Added by PPMD on November 5, 2013 at 2:00pm — 1 Comment

Identification

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 …

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Added by PPMD on November 4, 2013 at 3:24pm — 1 Comment

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