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All Blog Posts (1,773)


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
Wave Life Sciences Initiates Clinical Trial for Lead Program in Duchenne

Wave Life Sciences Ltd. announced the initiation of a global Phase 1 clinical trial for WVE-210201 in Duchenne patients amenable to exon 51 skipping. PPMD is excited by the progress Wave Life Sciences has made in exon skipping. While advances have been made in exon skipping, especially recently, there is more to explore within this technology. We…

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Added by PPMD on November 6, 2017 at 9:09am — No Comments


Staff
Sarepta & Nationwide Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne

More good news in gene therapy! Today, Sarepta Therapeutics announced that Nationwide Children’s Hospital has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for the micro-dystrophin gene therapy program. The Phase 1/2a clinical trial, now open to enrollment and scheduled for initiation by mid-November, is designed to assess the safety and tolerability of AAVrh74.MHCK7.micro-Dystrophin in individuals…

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Added by PPMD on November 6, 2017 at 8:30am — No Comments


Staff
Action Alert: Proposed Senate Tax Plan & the Direct Impact on Our Community

Call & Email Your Senators Today!

Yesterday, the Senate released the Tax Cuts and Jobs Act which includes many issues that we feel an obligation to alert you to.

 

While PPMD does not typically engage in legislation related to tax reform, this proposal (being referred to as…

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Added by Annie Kennedy on November 3, 2017 at 10:00am — No Comments


Staff
Sarepta Therapeutics & Nationwide Children’s Hospital Announce FDA Clearance of the IND Application for the GALGT2 Gene Therapy Program

Once again, PPMD is excited to see industry, patient groups, and research partnering, as today Nationwide Children's Hospital and Sarepta Therapeutics announced the achievement of an important milestone in…

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Added by PPMD on November 3, 2017 at 8:39am — 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

My Journey on the PAAC

I decided to join the PAAC after Parent Project’s 2015 Annual Connect conference in Washington, D.C. Annie, along with the 2015 PAAC members and a few other adults with Duchenne, hosted a pre-conference meeting for teens and adults living with Duchenne. When listening…

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Added by Colin Werth on November 2, 2017 at 2:21pm — 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…

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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
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

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