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No Exon Left Behind

Posted by Sharon Hesterlee on July 21, 2014 at 10:15am 6 Comments

I'm often asked by parents to explain exon skipping. In fact, at last month's 20th Annual Connect Conference, after we announced a grant awarded to Dr. Judith van…

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Dawn Steele replied to Dawn Steele's discussion Results in & deflazacort
"Thanks Donna. I spent $125.00 for 300 6mg tablets including shipping."
1 minute ago
Donna replied to Dawn Steele's discussion Results in & deflazacort
"Be careful on the shipping charges on the deflazacort.  The first vendor we went through charged us almost 50% of the cost of the deflazacort (might have been more) - it was insane.  Masters Marketing (& Julie Emms) has a pretty darn…"
33 minutes ago
Moein replied to Moein's discussion Does Idebenone have something to do with behaviours and cognitive issues?
" Some improvement has been detected but not that much! I am not sure if it really help lungs and heart since my son is okay until now. He was on 900 mg/day"
55 minutes ago
Profile IconParent Project Muscular Dystrophy via Facebook
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Care of the heart in Duchenne is not a simple business. Fortunately, more than 30 representatives from the cardiac care, cardiac research, the National Heart Lung and Blood Institute (NHLBI) and the FDA agreed, and were gracious enough to give two days of their very busy lives to discuss just these issues in a recent meeting convened by PPMD. Read more about the incredible discussions that took place as well as next steps based on the interest of the FDA.…

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Rahul Deshpande replied to Moein's discussion Does Idebenone have something to do with behaviours and cognitive issues?
"I read that Idebenone is still in clinical trials. Have you seen any improvement in your child by using Idebenone."
11 hours ago
David replied to Amrit's discussion Test for evaluating learning disablity
"I was in your shoes 10 years ago. IEPs are hard work, no test will give you more than a glimpse in time at best. Took him 5 years to shake the 'mild mental retardation' label, the result of a similar test. But my son is now pulling Bs in…"
14 hours ago
Rosalie replied to Amrit's discussion Test for evaluating learning disablity
"Hi My son has also been struggling in school and testing provided by district has not shown anything significant.  When I was in CCHMC a few weeks ago they suggested a Developmental Pediatrician.  So that is where we are headed.  And…"
22 hours ago
Dawn Steele replied to Dawn Steele's discussion Results in & deflazacort
"Thank you everyone for your comments.  I actually went forward with ordering Deflazacort from Northwest Pharamacy.  I did receive the medication today shipped from SwissPost but show it was manufactured by Lupin.  Which is an India…"
23 hours ago
Profile IconParent Project Muscular Dystrophy via Facebook
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BREAKING NEWS! We are thrilled to announce that moments ago the Senate HELP Committee approved the #MDCAREAct. This is another huge step in the right direction for our bill and follows recent action by the House Energy and Commerce Committee. We are getting closer to our goal of updating the #MDCAREAct Amendments! Stay tuned for continued updates and thank you for all of your hard work! Strength Happens Together! …

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Andrew Kerr replied to Rahul Deshpande's discussion Deletion of exon 44 in the group Deletions
"Sarepta is working on an exon skip of 45 and I believe they stated in a call that they hoped to start trials late this year."
23 hours ago

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It's a big day for the #MDCAREAct as the Senate HELP committee takes up our bill. We are so grateful to all our advocates for reaching out to your members. THIS JUST IN: We received great news that the HELP Committee Chairman Senator Tom Harkin has signed on an official cosponsor! Momentum continues to build for the critical legislation, but there's still work to be done. It is not too late if you haven't acted yet, it will only take one minute!…

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In a newly released white paper by Siren Interactive, produced from input by rare disease patient advocacy group leaders and patient opinion leaders, including PPMD Founding President & CEO Pat Furlong, Siren examines the issues around compassionate use and the need for proactive approaches by industry to both developing policies and communicating those policies to all interested stakeholders.…

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Sabine Wedel is now a member of PPMD Community
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Rahul Deshpande added a discussion to the group Deletions
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Deletion of exon 44

It seems almost 10% of DMD cases are due to deletion of exon 44. But the trials for exon skipping for 44 deletion is still in very early phase. Anyone having update on this. See More
yesterday
 
 
 

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