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All Blog Posts Tagged 'PPMD Blog' (664)


Staff
Two simple words...THANK YOU!

We not only met our goal, we surpassed it!





We could not be more overwhelmed by the incredible response to our holiday campaign. In many ways, with a therapeutic pipeline full of promising treatments, it feels like the future of Duchenne therapy is here. Thanks to this community, we head into 2017 with…

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Added by Pat Furlong on January 4, 2017 at 3:47pm — No Comments


Staff
What You Need to Know about Duchenne & Viral Gastroenteritis (That Horrible Stomach Bug!)

Viral gastrointestinal (GI) viruses are no fun for anyone, but they are especially worrisome for a person living with Duchenne muscular dystrophy. GI viruses affect the GI track – the stomach and intestine (i.e., ‘gut’) – resulting in abdominal pain/discomfort, nausea, vomiting, intestinal…

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Added by Kathi Kinnett on January 4, 2017 at 10:30am — No Comments


Staff
Make my parents pay--48 hours left!

We’re so close to our goal to fund CRISPR/Cas9!…

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Added by PPMD on December 29, 2016 at 9:30am — No Comments


Staff
This could help me--and everyone with Duchenne

Your donation will be DOUBLED. Now is the moment to give.…

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Added by PPMD on December 22, 2016 at 9:12am — No Comments


Staff
DOUBLE your investment in the future

Help us raise $250,000 by 12/31!…



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Added by Abby Bronson on December 20, 2016 at 12:00pm — No Comments


Staff
Understanding the Path to Access – Resources for the Community

We have all had frustrations getting healthcare paid for, whether it is getting access and coverage for appointments, procedures, equipment, and/or medications.

Coverage is especially difficult when new medicines or procedures are recommended. Parents,…

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Added by PPMD on December 19, 2016 at 12:30pm — 1 Comment


Staff
I Will Match Your Gift

Help us raise $250,000 to fund CRISPR/Cas9.



After a few months of relative quiet, noise has returned to the Killian house in full force! For the holidays, all five kids are back under one roof with a revolving door of friends and endless groceries. Even with all the chaos that entails, this is my favorite time of…

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Added by PPMD on December 13, 2016 at 9:38am — No Comments


Staff
New Paper Further Validates Need for Annual Cardiac MRI Surveillance in Duchenne

When PPMD convened the Contemporary Issues in Duchenne Cardiology meeting with National Heart, Lung, and Blood Institute (NHLBI) in July 2014, there were several recommendations.

The strongest recommendations included the use of cardiac…

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Added by Kathi Kinnett on December 8, 2016 at 4:00pm — No Comments


Staff
PPMD Is Seizing This Moment to Explore the Potential of CRISPR/Cas9 in Duchenne

It is the holidays – time for giving and laughter and hope. Hope comes in many forms, but I would imagine that for many reading this blog, hope comes in the form of a cure for Duchenne.  

Could CRISPR/Cas9 offer hope for treating Duchenne? 

As background, CRISPR/Cas9 is a…

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Added by Abby Bronson on December 8, 2016 at 3:30pm — No Comments


Staff
How PPMD’s Certified Duchenne Care Centers Are Improving Quality & Access to Care

When I joined PPMD in 2012, care for Duchenne was all over the map. 

 

Sadly, there were over 150 clinics spread across the US, but there was no oversight of these clinics – the community did not know who was providing care, the level of care being provided or whether anyone at…

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Added by Kathi Kinnett on December 8, 2016 at 3:02pm — No Comments


Staff
BREAKING NEWS: 21st Century Cures Crossed Over the Finish Line!

The Duchenne Community’s Voice Was Essential Every Step of the Way & Contains PPMD’s Provision to Strengthen Patient Focused Drug Development Efforts



To everyone in our PPMD community who called, reached out, emailed, and met with your elected…

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Added by Annie Kennedy on December 7, 2016 at 2:30pm — No Comments


Staff
PPMD’s #EverySingleOne Tour: Durham

Duke University marked our final stop on PPMD’s 2016 Every Single One Tour, held on November 2. We were thrilled to be holding another tour stop at one of…

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Added by Ryan Fischer on December 7, 2016 at 11:04am — No Comments


Staff
Light up the Lines!! Urge Your Senators to Vote YES on 21st Century Cures!

We need you to call your two US Senators TODAY with one simple message – VOTE YES ON 21st CENTURY CURES!!

 

With just days remaining in the current Congress, this bill represents the extraordinary efforts of our PPMD community’s work and outreach with…

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Added by Ryan Fischer on December 5, 2016 at 10:07am — No Comments


Staff
Your gift to CRISPR will be Matched!

We have so much momentum. Let’s seize this moment.





First of all, let me say THANK YOU! Your gift to PPMD on #GivingTuesday helped us surpass our goal, and we could not be more grateful. It is a wonderful way to kick off our holiday season—and we wanted to make sure you were among the…

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Added by Pat Furlong on December 1, 2016 at 9:30am — 1 Comment


Staff
A truly thankful moment

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Added by Pat Furlong on November 23, 2016 at 5:30pm — No Comments


Staff
Summit Enrolls U.S. Patients into Phaseout DMD, a Phase 2 Clinical Trial of Ezutromid in Duchenne

Summit Therapeutics plc announced today that it has enrolled its first patients at trial sites in the US into PhaseOut DMD, a Phase 2 clinical trial of ezutromid in Duchenne. The 48-week, open-label (meaning all participants receive ezutromid) Phase 2 trial is enrolling up to 40 boys total between their 5th…

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Added by PPMD on November 16, 2016 at 9:00am — No Comments


Staff
Duchenne Carrier Study at Nationwide Children’s Hospital

Frequently, I get calls from mothers/sisters/aunts of people living with Duchenne about issues related to being a carrier of Duchenne:



  • Should they have carrier…
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Added by Kathi Kinnett on November 14, 2016 at 11:00am — 2 Comments


Staff
CHMP Recommends Renewal of Translarna's™ Marketing Authorization Based on Continued Positive Benefit-Risk Assessment

PTC Therapeutics, Inc. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna(TM) (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients…

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Added by PPMD on November 11, 2016 at 9:30am — No Comments


Staff
2016 Flu Vaccination Recommendations

Everyone, six months of age and older, needs to be vaccinated against influenza (flu), and protecting people living with with Duchenne is especially important. A …

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Added by Kathi Kinnett on November 9, 2016 at 2:30pm — 2 Comments


Staff
Akashi Provides HT-100 Clinical Trial Update

In February, dosing and new patient enrollment in all cohorts of the HALO trial, a study evaluating the compound HT-100 in patients with Duchenne, was suspended after one of the patients in the trial, receiving 60μg/kg/d (the highest dose in the study), passed away after experiencing serious, life-threatening health issues. Since then, the company has been working with the…

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Added by PPMD on November 7, 2016 at 11:30am — No Comments

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