September 2017 Blog Posts (20)

You can still join the fight to end Duchenne!

As we head into the 10th Annual Coach To Cure MD weekend tomorrow, with emotions still running high from yesterday’s…


Added by Pat Furlong on September 29, 2017 at 10:06am — No Comments

Coming Full Circle: The Ataluren Ad Comm

Like any of you who participated in today’s Advisory Committee Meeting at the FDA for PTC Therapeutic’s ataluren, I am emotionally exhausted. Whether you participated by speaking during the Open Public Hearing, attending the Ad Comm on the FDA campus, submitting your written testimony, or streaming the meeting from home – I would imagine that you, too, are…


Added by Pat Furlong on September 28, 2017 at 5:30pm — No Comments

September 28: FDA Advisory Committee Meeting for Ataluren - Live Webcast Information

On Thursday, September 28, an FDA Advisory Committee will review ataluren for potential approval and use within our Duchenne community.

The Duchenne community is joining forces in a show of strength to…


Added by PPMD on September 27, 2017 at 6:42pm — No Comments

PPMD Continues Grant Supporting Clinic Liaisons for Certified Duchenne Care Center Program

PPMD is excited to announce that we will continue a grant program to help support our Certified Duchenne Care Center (CDCC) Program. The CDCC Program is a network of…


Added by PPMD on September 27, 2017 at 12:30pm — No Comments

Are You Ready for Coach To Cure MD This Saturday?

This weekend, nearly every televised College Football game around the country will have coaches wearing Coach To Cure MD armbands, tweeting about Duchenne, and encouraging fans to text a…


Added by PPMD on September 26, 2017 at 1:00pm — No Comments

Briefing Documents for Ataluren Ad Comm Released

The briefing documents for the September 28…


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

In an Emergency – Consider a Medical ID

Let’s say that Joe has Duchenne. If Joe breaks his arm and the EMT comes before Joe’s parent arrives, Joe may not remember to tell the EMT that: 1) he has Duchenne, 2) he is on daily steroids 3) he is allergic to penicillin and, in that painful scary moment, he may or may not remember his parent’s cell phone number. 


For some people, having a medical ID that…


Added by Kathi Kinnett on September 22, 2017 at 7:23pm — No Comments

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…


Added by Abby Bronson on September 21, 2017 at 12:00pm — No Comments

ACTION ALERT: Stop the Graham-Cassidy proposal in the Senate

The Senate is considering another effort to repeal the Affordable Care Act (ACA) before the end of the month. Last week, Senators Lindsey Graham (R-SC), Bill Cassidy (R-LA), Dean Heller (R-NV), and Ron Johnson (R-WI) released a new plan to repeal and replace the ACA. This proposal is commonly referred to as…


Added by Annie Kennedy on September 19, 2017 at 10:00am — No Comments

2017/2018 Flu Vaccination Recommendations

Everyone, six months of age and older, needs to be vaccinated against influenza (flu) by the end of October, if possible. Protecting people living with Duchenne is especially important. A 2005 study done by…


Added by Kathi Kinnett on September 18, 2017 at 10:30am — No Comments

Santhera Receives Negative Opinion from the CHMP for Raxone® and Intends to Appeal this Opinion

Santhera Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion for its Type II extension application for Raxone® (idebenone) in Duchenne. Santhera plans to appeal the opinion and will seek a re-examination by the CHMP. We appreciate…


Added by PPMD on September 15, 2017 at 8:00am — No Comments

PPMD Designates Nemours/Alfred I duPont Hospital for Children a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named Nemours/Alfred I duPont Hospital for Children (Nemours) in Wilmington, Delaware our 17th Certified Duchenne Care Center.

PPMD’s Certified Duchenne Care Center Program, which supports standardized, comprehensive care and services for all…


Added by PPMD on September 14, 2017 at 8:30pm — No Comments

PPMD Lends Leadership to NCATS Rare Disease Research Toolkit Development

There are some days when I feel like our community is in its infancy. When I look at where we are in terms of access, therapeutic options, combinations, care implementation…

But more often than not, I am overwhelmed with gratitude for the infrastructure and resources available to our Duchenne community. The rich collaborations and strong…


Added by Annie Kennedy on September 13, 2017 at 5:00pm — No Comments

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. …


Added by Kathi Kinnett on September 13, 2017 at 4:30pm — No Comments

MD Community Moves Forward ICD Code Nomination for Duchenne/Becker

Paperwork – oh the paperwork!

Each time you visit a physician’s office or hospital, we leave with piles of forms and papers detailing the visit. The providers seen, the prescriptions written, results from tests conducted, instructions for follow up, and more. And each of those pieces of paper includes numerous codes that allow for tracking…


Added by Annie Kennedy on September 13, 2017 at 10:00am — No Comments

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,…


Added by PPMD on September 12, 2017 at 9:30am — No Comments

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…


Added by Louie Lewis on September 11, 2017 at 9:56am — No Comments

The 2018 End Duchenne Tour – Coming to a City Near You!

PPMD is excited to announce next year’s lineup of areas we will visit as part of our End Duchenne Tour!

Combining each of the pillars that make up PPMD’s mission, the End Duchenne Tour brings updates on research, advocacy, and care to cities across the country, featuring a roster of leading experts in the…


Added by PPMD on September 8, 2017 at 5:30pm — No Comments

Sarepta Therapeutics Announces Positive Results from 4053-101 Study

Sarepta Therapeutics, Inc. today announced muscle biopsy results from its 4053-101 study, a Phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of golodirsen…

Added by PPMD on September 6, 2017 at 10:00am — No Comments

Roche Provides Community Update on BMS-986089 (RG6206)

Earlier this year, Bristol-Myers Squibb (BMS) announced it had entered into an agreement to license BMS-986089 (RG6206), an anti-myostatin adnectin in development for Duchenne muscular dystrophy, to Roche. Today, the Roche team has provided an update to our…


Added by PPMD on September 5, 2017 at 9:30am — No Comments

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