April 2014 Blog Posts (47)


Staff
My BRAVE Story: It has been a long and slow progress

MY STORY COMES FROM TO YOU FROM INDIA,HYDERABAD,A.P WHERE THERE IS HARDLY ANY AWARENESS OF THE DREADED DISEASE CALLED DUCHENNE MUSCULAR DYSTROPHY . MY SON YASH BAKSHI AT 3 YEARS WAS DIAGNOSED WITH DMD WITH EXON 49,50 MISSING AFTER MUSCLE BIOPSY TEST.. OBVIOUSLY MY WORLD TURNED UPSIDE DOWN AND FOR A LONG TIME I WENT OVER THE QUESTIONS WHY ME & WHY HIM.. THEN OUR WORRY TURNED TO OUR 9 MONTH OLD 2ND SON, WHAT IF HIM TOO.. WITH GOD'S GRACE HE WAS FREE FROM THE DISEASE AFTER MANY…

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Added by PPMD on April 21, 2014 at 12:31pm — No Comments


Staff
My BRAVE Story: I beg the FDA to intervene and act quickly for these children

My name is Mindy Cameron and my son Christopher was diagnosed with Duchenne in 2003 when he was two years old. He does not have one of the more common gene mutations and his disease is progressing very typically, according to what we know from Duchenne natural history. I find that we often hear of the boys who defy the natural history of Duchenne. We marvel at the young men who still walk at age 17 and beyond, we talk of 30 somethings who hold down jobs and even have families, we…

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Added by PPMD on April 21, 2014 at 12:00pm — No Comments


Staff
My BRAVE Story: Time is something that WE do not have, Duchenne is a ticking time bomb

Dear FDA Officials, My name is Dreama Whitlock, and I am writing to you to request the accelerated approval of safe and effective treatments for those living with Duchenne Muscular Dystrophy. Duchenne is 100% fatal. In 2011, my grandson Riley (5 at the time) was diagnosed with Duchenne. The medication Eteplirsen "will not" help Riley, however it could open the door to new treatments that would benefit him. Riley has a point mutation, not a deletion or duplication. The end result…

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Added by PPMD on April 21, 2014 at 12:00pm — No Comments


Staff
My BRAVE Story: I am lucky

I am the grandmother of 8. 1 of my grandsons has to have his kidneys removed and go on dialysis the nurses tell him is so upbeat about it why. He said I have a treatment my cousin does not. I am lucky

Added by PPMD on April 21, 2014 at 11:56am — No Comments


Staff
My BRAVE Story: It is a progressive disease

My grand son have DMD he is 9 year old and can not walk very much and it is a progressive disease, we are Argentines, I can not write very well in English. All the patients with this disease need URGENT same treatment for a good life. thanks

Added by PPMD on April 21, 2014 at 11:49am — No Comments


Staff
My BRAVE Story: I know our government is more powerful than a disease, I wish they could show us that power and END Duchenne

My son is 7. He should be in school, invited to birthday parties from his peers, playing sports and riding his bike with the kids down the street. He can't because Duchenne has stole all of that from him. Nicholas has a genuine smile that can light up a room and a twinkle in his eye that could melt any moms heart but he crumbles in social settings, Duchenne affects his body in so many ways, he also has autism that may be lack from the Dystrophin gene in the brain like so many…

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Added by PPMD on April 21, 2014 at 11:47am — No Comments


Staff
My BRAVE Story: There should be more extensive approaches to therapy for children with this disease

My son, Ryan, was diagnosed with Duchenne Muscular Dystrophy on May 29, 2008 at the age of two and a half. He has a frameshift mutation of exon 38. We knew shortly after he was born that something was wrong. He wasn't reaching the milestones like a typical child. After many tests, tears, and false hopes we were given the diagnosis we feared the most, Duchenne Muscular Dystrophy. In addition to his DMD, Ryan also faces the challenges of autism, suspected to come about as a result…

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Added by PPMD on April 21, 2014 at 11:37am — No Comments


Staff
My BRAVE Story: Please help our Duchenne families

My son Owen was diagnosed with Duchenne Muscular Dystrophy in October of 2012. It was a diagnosis we never expected to receive and also had never heard of this disease and knew nothing about it. When we were told "There is no cure for this disease" and i asked "will he die from this" our doctor at the time said "yes" its a feeling no mother can ever explain but its like your heart is being ripped out of your chest.…

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Added by PPMD on April 21, 2014 at 11:30am — No Comments


Staff
My BRAVE Story: Say YES to a life for our boys, say YES to Eteplirsen

We're the May family. My name is Jessica, I met my husband, Mark, in Afghanistan in 2009. We have a 2.5 year old son named Markie, he has Duchenne. We started noticing delays within the first 6 weeks, but every child is different so we didn't think much of it. It wasn't until Christmas of 2011, when Markie was 5 months old, that we really started to worry. Most people told me not to worry and that slight delays were normal, they said boys are sometime slower anyway. I still…

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Added by PPMD on April 21, 2014 at 11:30am — No Comments


Staff
My BRAVE Story: These children and young adults deserve to have a good long life as much as everyone else

My son Brent was diagnosed with Duchenne Muscular Dystrophy when he was 2 months old, he is 6 years old now. My 15 year old nephew Jt also has this disease. This disease is progressive, weakening the muscles and it's eventual result is an early death. It has been hard coping with my son and nephew having this. As time passes, they are weakening more. Although my…

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Added by PPMD on April 21, 2014 at 11:30am — No Comments


Staff
Sarepta Therapeutics Announces Plans to Submit New Drug Application to FDA for Eteplirsen for the Treatment of Duchenne Muscular Dystrophy by Year End 2014

Great investor call from Sarepta this morning on the heels of the news about positive signals from the FDA for submitting a new drug application! We are speaking with the company this morning and will release a detailed statement this afternoon. Congratulations to everyone!







Sarepta's Press…

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Added by PPMD on April 21, 2014 at 9:30am — 1 Comment


Staff
My BRAVE Story: These families and children deserve as much help as possible in every way shape and form

My nephew Bradley A. Butler was diagnosed when he was five with duchenne. We knew from much early on that something was wrong. He was delayed on alot of growing milestones like crawling, standing,walking an he was never able to run. I was only 12 when he was born an so i was there an saw first hand how hard of a process it was to find out what was wrong an the hopeless feeling of only having 20yrs with this special lil man.

Bradley only survived 6weeks before his 16th bday.…

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Added by PPMD on April 15, 2014 at 11:46am — No Comments


Staff
My BRAVE Story: We want a cure!

Duchenne Muscular Dystrophy, you have rocked our world! We were given this beautiful baby boy to love, cherish, and enjoy him for many years. Instead, you have taken over our wonderful young man. You did not even hold out for a little while. From birth you were there interrupting his life. You did not allow him to run, jump, skip,nor all the other fun things a child should enjoy. How do you answer a child when he asks the questions: "Why" "Will I ever" "How bad, is bad enough"…

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Added by PPMD on April 15, 2014 at 11:43am — No Comments


Staff
My BRAVE Story: Give them a chance to live longer, fuller lives

My son, Elijah, is 9 years old. His favorite thing to do is help me in the kitchen. He particularly enjoys baking. Other things he likes to do include dancing, drawing, and crafts.

When he was four years old, he was diagnosed with DMD. As the diagnosis was not new to me; I was devastated. I knew that it was a slow death sentence. I have a family history and…

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Added by PPMD on April 15, 2014 at 11:30am — No Comments


Staff
My BRAVE Story: We take this disease one day at a time for that is all we can do

Our son, Matthew, was diagnosed with Duchenne at 18 months old. We had never heard of DMD, no one in our family had it. After researching it we cried for our son, our dreams for him, his dreams he would have not be able to fulfill. Now he is 14 and uses a wheelchair full time. We take this disease one day at a time for that is all we can do. At this age his arms…

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Added by PPMD on April 15, 2014 at 11:30am — No Comments


Staff
My BRAVE Story: Please give us hope. Please give us more time. Please support treatments for our boys.

Kaleb and Liam are two amazing boys with smiles that are contagious. They are five and eight years old. When you look at them they look happy and healthy just like any other boys their age. Running, playing, exploring, and laughter are the things that they do best. Although they appear fine at first glance, if you take a moment to really watch you may notice there is something that is progressively slowing them down. As babies the boys developed normally. There wasn't much…

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Added by PPMD on April 15, 2014 at 11:30am — No Comments


Staff
My BRAVE Story: No Mom should ever have to hear the words I did

My life turned upside down on August 10th 1989.I was 8 month pregnant with my 3rd child.I took my then 4 yr old son to UW Madison to see the pediatric neurologist. .He look at my son and said he has Duchenne Muscular DYSTROPHY.... No clue what that means... He then told me my son would not live to be 15 maybe 20..Shock....my heart stopped.. Then I was told your child you are carrying may also be affected... Great..One month later I had a baby boy...He to has Duchenne...Now hope he…

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Added by PPMD on April 15, 2014 at 11:30am — No Comments


Staff
Success on Funding Letter, Time to re-engage on the MD-CARE Act



Thanks to your efforts, we closed the House and Senate funding letters with the support of 30 House members and 12 Senators. Great work! We appreciate everyone who sent emails and made calls to their members of Congress.

The members below collectively sent a strong message to the House and Senate appropriations committees…

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Added by Ryan Fischer on April 15, 2014 at 9:51am — No Comments

By my little jacob

I'm as small as a chair.

I'm as strong as a gorilla.

I'm as quiet as a snake.

I'm as wiggly as a worm.

I'm as fast as a tortoise.

I'm cuter than a button.

I'm more loving than a puppy.

I'm soft like a pillow.

I'm perfect just like I am.

When a 8 year old can write this and be happy with the way they are why cant adults be that way to? We may not think that our dmd boys are perfect and we may try to find ways to fix them…

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Added by danielle on April 14, 2014 at 10:03pm — 1 Comment

what is there to do now?

We went to the mda clinic on the first. The doctor there told us that my son doesnt need to wear his AFOs (leg braces). This made my son and myself very happy because he hates wearing them. Well after being told this we went to the pt and she informed us that he needs to wear them regaurdless of how strong his ankles are because with him being in his wheelchair his heel chords will get tight and he wont be able to take a step. After my son heard this he got scared he says im scared not to be…

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Added by danielle on April 14, 2014 at 9:57pm — No Comments

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