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October 2017 Blog Posts (12)


Partner Information
Understanding Duchenne: Preventing CGTF & Fibrosis

In many diseases, the development of fibrosis in muscle, or any tissue, is the result of a reactive, or faulty tissue repair process involving many factors.



In Duchenne, fibrosis is present in skeletal muscles at a very early age and is one of the major factors…

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Added by PPMD Partner Information on October 17, 2017 at 1:00pm — No Comments


Staff
Your DuchenneConnect Registry Data is Advancing Research! New Publication

Do you ever wonder what happens to all the data we collect in the DuchenneConnect Registry? Ever wonder why we regularly ask you to update your registry account?

How your data is…

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Added by Ann Martin on October 16, 2017 at 10:29am — No Comments


Staff
Gene Transfer in the Spotlight

On September 28 and 29, I had the privilege of attending an in-person Wellstone Center meeting that Dr. Eric Olson and his Wellstone team at UT Southwestern Medical Center hosted in Dallas, TX. The breadth and depth of what the Wellstone Centers have worked on over the years…

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Added by Abby Bronson on October 11, 2017 at 12:30pm — 2 Comments


Staff
News from 2017 World Muscle Society: Part 2

Annie Kennedy and Kathi Kinnett from the PPMD team recently attended the 22nd International Annual Congress of the World Muscle…

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Added by PPMD on October 9, 2017 at 12:30pm — No Comments


Staff
83 International Experts Submit Letter to FDA Encouraging Approval of Ataluren

PPMD applauds the group of 83 international experts with experience caring for Duchenne patients around the globe who have written a letter to the FDA encouraging approval of…

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Added by PPMD on October 9, 2017 at 10:36am — No Comments


Staff
News from 2017 World Muscle Society: Part 1

 

Annie Kennedy and Kathi Kinnett from the PPMD team are attending the 22nd International Annual Congress of the World Muscle Society…

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Added by PPMD on October 6, 2017 at 1:30pm — No Comments


Staff
Steroid Dosing & Duchenne: FAQs

Steroid dosing in Duchenne can be a confusing topic – what is enough, what is too much, why weekend doses are higher. With these frequently asked questions I’ve heard from the community…

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Added by Kathi Kinnett on October 5, 2017 at 3:00pm — No Comments


Staff
2017 World Muscle Society: Highlighting Progress in Duchenne

“WMS - What’s the buzz?”

 

It has been many years since I’ve had the opportunity to attend the World Muscle Society meeting and wow how things have changed! While PPMD has strong representation here every year, this year I have…

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Added by Annie Kennedy on October 4, 2017 at 1:00pm — No Comments


Staff
Catabasis Reports Positive Results from Open-Label Extension of MoveDMD Trial with Edasalonexent

Catabasis Pharmaceuticals, Inc. today reported new positive efficacy results following 24 and 36 weeks of treatment with 100 mg/kg/day oral edasalonexent in…

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Added by PPMD on October 4, 2017 at 10:30am — 1 Comment


Staff
Letter from PTC Therapeutics to the Community Following FDA Ad Comm

PTC Therapeutics President and CEO, Stuart Peltz, Ph.D has shared a letter with the Duchenne community following last week's…

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Added by PPMD on October 3, 2017 at 9:30am — No Comments


Staff
Webinar Q&A: Gene Therapy Part 2 – Nationwide Children's Hospital

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…

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Added by Abby Bronson on October 2, 2017 at 1:30pm — No Comments

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