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Exon 17 Duplication

Started by ruben lengler in Research on Tuesday. 0 Replies


Started by Azucena Lopez de Nava in Research. Last reply by Antonio Castro on Monday. 3 Replies

Emflaza (aka deflazacort) Approvals

Started by Keith & Jill VE in General Discussion. Last reply by Keith & Jill VE on Saturday. 4 Replies

Exon Duplications

Started by Sai Chand. Gurujala in Research Sep 11. 0 Replies

Latest Activity

PPMD updated an event

PART 2: Gene Therapy Webinar 9/6 – Nationwide Children's Hospital at Online Webinar - Eastern Time Zone

September 6, 2017 from 2pm to 3pm
This fall, PPMD is presenting a gene therapy webinar series with companies and institutions who are developing therapies for Duchenne that are commonly referred to as gene therapy, including micro-dystrophin and CRISPR/Cas9. The second webinar in this series took place on Wednesday, September 6 at 2 PM ET, featuring Dr. Jerry Mendell of Nationwide Children’s Hospital who discussed…See More
13 hours ago

Abby Bronson posted a blog post

PPMD Research Update: September 2017

As you know by now, September is Duchenne Action Month. And while the idea behind this campaign is to encourage our community to take action, the momentum in Duchenne research has never felt more active...and…See More
19 hours ago
Sai Chand. Gurujala replied to Dalibor Randjelovic's discussion Is it dr Kevin Flanigan`s research hope for our boys with duplication? in the group Duplications
"Sir, Any updated status on Duplication research. Please share the update. Thank you."
20 hours ago
Sai Chand. Gurujala commented on PPMD's group Duplications
"Hi All, Clinical research is going on Deletions. Anybody has any idea on Duplication's research. Serepta Pharma working on 40-53 Deletions. Does Gene Therapy is applicable for Duplication's as well. Please share . Thank you"
20 hours ago
sulthana shaik posted a discussion

Exon 45 skipping/ exon 51 skipping, Help to make a hard decision !!.PLEASE HELP.

HiWe are planning to participate in exon 45 skipping trail, for our 12 year old son.Is any one already enrolled in the exon skipping  trail for either 51/45/53, Can you please share your experience. Struggling to decide if it will be the best decision for our son.Are there huge risks?See More
ruben lengler posted a discussion

Exon 17 Duplication

Hi my son has duplication (Exon 17) anybody with that specific Exon 17 duplication?See More
Profile IconHirotami Ohara, ruben lengler, Carrie and 1 more joined PPMD Community

PPMD posted blog posts
Sep 15

Annie Kennedy posted blog posts
Sep 13

Kathi Kinnett posted a blog post

PPMD’s #EverySingleOne Tour: Charleston, WV

We kicked off our fall Every Single One Tour stops in West Virginia this past weekend! We appreciate all of the families, speakers, and volunteers that spent the day with us in Charleston. Psychosocial Issues in Duchenne…See More
Sep 13
Akram Dergali is now a member of PPMD Community
Sep 13
Regina Reidenberg is now a member of PPMD Community
Sep 12

PPMD posted a blog post

Reminder: Deadline to Submit Written Comments for Ataluren Ad Comm is Thursday, September 14

The deadline to submit a written comment for the FDA Advisory Committee Meeting on ataluren is this upcoming Thursday, September 14th. To make a comment,…See More
Sep 12

Kathi Kinnett commented on Kathi Kinnett's blog post Important New Duchenne Steroid Protocol: Download the "PJ Nicholoff Steroid Protocol"
"Tia - a stress dose of steroids, if your son had been using daily steroids for more than 2 weeks, would absolutely have been advised.  However, this sounds like the sysmptoms of a fat embolism.when-parents-are-informed.  When patients have…"
Sep 11
Sai Chand. Gurujala posted a discussion

Exon Duplications

Hi All,Clinical research is going on Deletions. Anybody has any idea on Duplication's research.Serepta Pharma working on 40-53 Deletions.Does Gene Therapy is applicable for Duplication's as well. Please share .Thank youSee More
Sep 11
Louie Lewis posted a blog post

The Muscle Gain Truth No Fail System Review

The dynamics of weight loss are intriguing, in take a look at the many because they came from desire to shed some weight in comparison to the small number who do. You would think with all the diet plans and dietary supplements, that weight problems would go away. But that hasn't came about, so something must be wrong. The sheer number of programs and products out there helps it to be difficult to find out what is effective and what is not. And it is far from as if one solution is effective in…See More
Sep 11

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