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March 2014 Blog Posts (30)

PPMD Awards $100,000 in Exploratory Grants

PPMD is thrilled to award two exploratory grants as part of our ongoing grant program. Peter Arthur of the University of Western Australia was awarded $50,000 to finish testing a molecule called procysteine in animal models in preparation for a human clinical trial. Procysteine is an antioxidant that has…

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Added by Sharon Hesterlee on March 25, 2014 at 11:42am — 2 Comments


Staff
Certified Duchenne Care Center Program

Right now, there are more than 10-12,000 young people diagnosed and living in the United States with Duchenne. These young people are being cared for by clinics both near and far. Every one of them deserves the best care and treatment possible. Families want care and treatment to be transparent; they want to know who is delivering care to their child, what comprehensive care looks like, and which centers are delivering that care. For this reason, a group of experts agreed to give families…

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Added by Kathi Kinnett on March 20, 2014 at 9:00am — 2 Comments


Staff
3 Actions You Can Take to Tell the FDA to Speed the Approval of New Therapies for Duchenne

As a community, we must continue to demand that the FDA use all available tools given to them by Congress to speed the approval of new therapies to all who so desperately need them. The voices of patients and caregivers are critical to the process of drug development and we believe the message is being heard.

 

Here are 3 simple actions you can take to support & amplify that message:

 

  1. Urge your…
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Added by Ryan Fischer on March 19, 2014 at 12:10pm — No Comments


Staff
My BRAVE Story: One step at a time

One step at a time... When I hold his little hand in mine it reminds me of the day when he held my hand and took those precious little steps one at a time, he discovered the joy of walking, the joy of independence totally unaware of what lies ahead. Today Duchenne is like an engraved finger print in life that ceases to erase, reminding us day in and day out of its presence, the challenges it throws in our path, the pain it causes and the emotional drain that it brings. There are…

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Added by PPMD on March 19, 2014 at 9:30am — No Comments


Staff
My BRAVE Story: Duchenne was an unwelcome guest in our lives

Years ago,when Daniel was almost five, we received the news that he had Duchenne muscular dystrophy. We knew nothing about Duchenne at the time, but now that Daniel has just passed his 20th birthday, we have become reluctant experts, and his sister has become an outspoken advocate for people with disabilities. At five, Daniel walked funny--so said the other kids. At seven, he bloated up due to the steroids. At eight, he began using a wheelchair--too many near stampedes in the…

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Added by PPMD on March 19, 2014 at 9:30am — No Comments


Staff
My BRAVE Story: We have tried and tried

I am a mother of four; 2 boys with DMD and 2 girls. My elder son,Mohammed, is 20 years now and Yossif is 16 years old.They have been suffering from DMD for over 14 years. I am trying my best and so does their father. But we are getting older day by day.If this new medication can help, please I would like to be with the other families who are in the same boat just like us. We have tried and tried and are really tired physically and financially. We are Egyptians and live in…

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Added by PPMD on March 19, 2014 at 9:30am — No Comments

Simple Actions: Real data can drive change

For years our community has been making noise so that the scientific and medical communities would listen and focus on Duchenne. They're listening – now we need to talk! Filling out a survey or doing an interview about your family's experiences with Duchenne creates important data. This data…

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Added by Holly Peay on March 18, 2014 at 9:30am — No Comments


Staff
Prosensa Announces Drisapersen 48-Week Data and Recent Corporate Developments

Prosensa just released their 48-week data for drisapersen, as well as an update on recent corporate developments. Both press releases are included below.



PPMD has been talking with Prosensa and will be hosting a webinar with UPPMD…

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Added by PPMD on March 18, 2014 at 9:00am — No Comments


Staff
My BRAVE Story: He is planning on being an independent adult with a full life expectancy

My son is Alexander. Alexander is 15 and a half, and I should be giving him driving lessons, but I can't. He has been confined to a wheelchair since he was 13 and cannot work the pedals. He now also lacks the upper body strength to operate a hand controlled vehicle. He works hard to take his medication, do his therapy, and takes school seriously. He is planning on being an independent adult with a full life expectancy. However, that will be for nought if the FDA does not grant…

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Added by PPMD on March 17, 2014 at 2:30pm — No Comments


Staff
My BRAVE Story: We are in a race against time

In September 2006, we got the news that our son, Eddie, had Duchenne Muscular Dystrophy. He was just a four year old seemingly healthy and happy little boy. My husband and I immediately started to research this disease, the progression and any possible treatments. It was hard for us to read what the future was going to be like for our son. Once we digested the reality that there was no cure and no treatment all we had was hope. We said to each other there is progress being made in…

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Added by PPMD on March 17, 2014 at 2:30pm — No Comments


Staff
My BRAVE Story: We need ACTION!

If I were sitting in front of the FDA I would ask if they had children. I would ask what their hopes and dreams were for their kids. Then I would ask, how they would feel knowing their child would not celebrate their 30th birthday. I've been on this journey with Andrew for 10 years. Ten years of creating a "new normal" after every loss. Ten years of watching a beautiful smart healthy young man grow so weak he can't do anything for himself. I would tell them of the heartache and…

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Added by PPMD on March 17, 2014 at 2:00pm — No Comments


Staff
My BRAVE Story: Each day you wait, you kill them faster

Six years ago when our son was diagnosed with Duchenne Muscular Dystrophy several doctors told us there would be as close to a cure as possible in two years. Obviously they were being optimistic. At that time, research on Duchenne had been in place well over 20 years.

Today well over…

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Added by PPMD on March 17, 2014 at 2:00pm — No Comments


Staff
My BRAVE Story: It was the most devastating news any parent could ever receive

My son Benedict was diagnosed in 2003 at 4 1/2 years old while I was 5 months pregnant with my daughter. It was the most devastating news any parent could ever receive. It was our worst nightmare to learn my son had a progressive and terminal illness with no treatments or cure available. It has been devastating to our entire family, especially my son. Our family has never been the same since.

My son lost his ability to walk at age 10. His heart function started declining at…

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Added by PPMD on March 17, 2014 at 9:38am — No Comments


Staff
My BRAVE Story: He wonders 'Why me?' all the time

My brother, Nabeel, is suffering form Duchenne Muscular Dystrophy. I have seen my brother's condition detriorate day by day and I have seen him struggle every single day for life. I have seen him smile the brightest smile despite knowing that there is no hope and no cure for his disease. I have seen him cry alone, at night, when everyone went to sleep. I have seen his grin stretching from ear to ear when I kissed his forehead. I have seen how scared he has been of being left…

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Added by PPMD on March 17, 2014 at 9:30am — No Comments


Staff
My BRAVE Story: A drug that could help him feels so close yet so far away

My beautiful son Connor: Is 9 years old Needs exon 53 skipped Can pass the six minute walk test Just got his first power chair (loves it). A drug that could help him feels so close yet so far away. I can taste it. I can pray. I can hope. Yet I find myself thinking if only he had been born now maybe there might be a drug available to help him when he needs it. Then, I think of everyone who was born ten years before my son. There were not the possibilities then that we have now. How…

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Added by PPMD on March 17, 2014 at 9:30am — No Comments


Staff
My BRAVE Story: My husband has chronicled some of the story of our son Jacob and his experiences with drisapersen

My husband has chronicled some of the story of our son Jacob and his experiences with drisapersen on the news website he publishes:

http://www.altadenablog.com/2012/10/i-was-a-jet-setting-guinea-pig-for-science.html…

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Added by PPMD on March 17, 2014 at 9:23am — No Comments


Staff
My BRAVE Story: Give him a fighting chance - that's all we ask

Give him a fighting chance - that's all we ask!!Felix is a twin, almost 3 years old. His brother stands 4inches taller. They are, by their own admission, best friends. Their 2 older brothers adore their two tiny tigers! As parents our lives shook and our hearts cracked when 8 months ago Felix was diagnosed with DMD. Each day there is a constant heartache that this horrendous disease will change all our lives forever. It is unbearable to think about. That he can't play football…

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Added by PPMD on March 17, 2014 at 9:00am — No Comments


Staff
My BRAVE Story: Our children are counting on us

Each night without fail, I wake up to waves of panic washing over me - panic over watching my precious 9-year-old son, Jake, deteriorate each day and my complete inability to do anything about it. Jake is following the typical progression of Duchenne. He has always struggled with his physical abilities, fatigue and inability to keep up with his peers. Over the past year, we've seen a definite decline in his overall strength. He's having much more difficulty with stairs, frequently…

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Added by PPMD on March 17, 2014 at 9:00am — No Comments


Staff
My BRAVE Story: Our boys want to live

Nikita is 9. He was diagnozed, when he was 4. Since that moment our life turned into before and after. Year by year he needed more assistance. It was hard to realize in one day, that all your dreams crashed, that your child will never run like others and will never be able to play his favorite football. Boys, affected by Duchenne become weaker and weaker day after day. They loose their muscles and we cannot stop it!

Please let our boys hope for the future normal life. Accepting the…

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Added by PPMD on March 17, 2014 at 9:00am — No Comments


Staff
My BRAVE Story: I have two sons with DMD

I have two sons with DMD. Elliott is now 10 and Henry is 7. You feel helpless as a parent as you watch them get older and lose ability and you can do nothing to stop it. We are blessed that both are functioning fairly independently, but we see decline in function as each year passes.

It is heartbreaking to see your child struggle with things we do without thinking- like unscrewing the cap off of a tube of toothpaste, sticking a straw into a juice box or clearing just one…

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Added by PPMD on March 17, 2014 at 9:00am — No Comments

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