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All Blog Posts Tagged 'PPMD Blog' (764)


Staff
Capricor Reports Significant Improvements in Duchenne Patients Treated with CAP-1002 Investigational Cell Therapy

PPMD is excited to learn that Capricor's investigational cell therapy, CAP-1002, is showing significant improvements in people with Duchenne. We look forward to learning more details about this therapy in a community webinar on Wednesday, November 29 at 1pm eastern. Details coming soon!

Read the release from…

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Added by PPMD on November 15, 2017 at 2:30pm — No Comments


Staff
PPMD Submits Community Sign-On Letter to FDA Supporting Approval of Ataluren

PPMD has been continuing to engage with both PTC and the FDA on behalf of the patient community regarding the regulatory review of ataluren.



Earlier this month, …

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Added by PPMD on November 13, 2017 at 1:00pm — No Comments


Staff
"My Journey on the PPMD Adult Advisory Committee"

Ever wished you could change the world – or at least your piece of it? Been looking for a way to engage a bit more? Have personal experience that you’d like to see be better reflected in national policy and program efforts?



Hear first-hand what…

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Added by Annie Kennedy on November 8, 2017 at 2:30pm — No Comments


Staff
SMA Gene Therapy Study: Their Success is Our Hope

Yesterday, the New England Journal of Medicine (NEJM) published “Single-Dose Gene-Replacement Therapy for Spinal Muscle Atrophy.” 

Spinal muscular atrophy…

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Added by Pat Furlong on November 2, 2017 at 9:54pm — No Comments


Staff
Update on Formal Dispute Resolution Process For Ataluren

On October 25, PTC Therapeutics shared the news that the FDA had issued a Complete Response Letter regarding their application for ataluren, noting that FDA had…

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Added by Annie Kennedy on November 2, 2017 at 9:30am — No Comments


Staff
PPMD’s #EverySingleOne Tour: Boston, MA

Boston made for a great location for our October Every Single One Tour stop. Nothing beats the northeast in the fall. Boston is home to a number of great Duchenne…

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Added by Ryan Fischer on October 30, 2017 at 2:00pm — No Comments


Staff
Calling All Adults with Duchenne/Becker – Join the 2018 PPMD Adult Advisory Committee!

Ever wished you could change the world – or at least your piece of it? Been looking for a way to engage a bit more? Have personal experience that you’d like to see be better reflected in national policy and program efforts?



PPMD is seeking adult members of our Duchenne/Becker…

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Added by Annie Kennedy on October 30, 2017 at 11:37am — No Comments


Staff
Letter from PTC Therapeutics to the Community Following FDA Decision

PTC has shared a letter with the Duchenne community in regards to the recent FDA decision. PTC will be holding a community call on Friday, October 27 at 12:00 PM EST to discuss next steps with the community. Please e-mail your…

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Added by PPMD on October 26, 2017 at 9:30am — No Comments


Staff
The Future of Ataluren: A Hard Day But Still a Path Forward

 

Today the FDA issued a Complete Response Letter regarding PTC Therapeutics’ application for ataluren. Today’s news is…

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Added by Pat Furlong on October 25, 2017 at 1:22pm — No Comments


Staff
Accelerating the Clinical Trial Process in Duchenne

Lengthy trials with variable results, based on endpoints such as the 6-minute walk test that can limit trial enrollment to ambulant individuals with Duchenne – this is today’s reality for clinical trials in this disease.

 

But progress is being made and PPMD is proud of our…

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Added by Abby Bronson on October 24, 2017 at 3:00pm — No Comments


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

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