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December 2015 Blog Posts (22)


Staff
Last chance to have your donation doubled!

This is it. We’re so close. We’re just $24,000 away from our goal of $200,000 to invest in early-stage research that can help the entire community.

And we’re also just a few short hours until our matching gift deadline. …

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Added by PPMD on December 31, 2015 at 8:00am — No Comments


Staff
Less than 48 hours left!

Two weeks ago I came to you with exciting news and a challenge: The Dumm family had stepped forward to offer a second match for this year’s holiday campaign, after a generous match from the Weismans.

Well here we are, less than 48 hours away from a New Year and just…

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Added by PPMD on December 30, 2015 at 8:00am — No Comments


Staff
My mom and dad will match your donation!

This is my favorite time of year. Not because of the presents or the new Star Wars movie—which by the way is AMAZING!—but because it feels like there is hope in the air.

A couple of weeks ago, when my parents finished listening to a webinar, they were so excited.…

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Added by PPMD on December 28, 2015 at 9:58am — No Comments

Nurturing Hope Through the Art of Nature (A Calender for a Cure)

I didn't deal well with my son's diagnosis of Duchenne, at first -- I took a selfish, cowardly, escapist approach, numbing my mind with sex, drugs, alcohol, cigarettes and other self-destructive…

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Added by David Stalling on December 24, 2015 at 3:08pm — 1 Comment


Staff
EMA Publishes Guidelines on the Clinical Investigation of Medicinal Products for Duchenne

The European Medicines Agency (EMA) has just published guidelines for Industry on the clinical…

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Added by PPMD on December 23, 2015 at 9:57am — No Comments


Staff
Please give now and we'll match your gift!

The holidays are an exciting time for our family—as I’m sure they are for yours. We’re grateful to be spending Christmas with our entire family. We take advantage of every holiday, every birthday, every special occasion to celebrate the strength of our family. And we never take for granted the time we have been given to be…

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Added by PPMD on December 22, 2015 at 3:00pm — No Comments


Staff
BioCentury Article: How BIO and PPMD plan to create a blueprint for patient preference studies

Earlier this month, PPMD announced a partnership with BIO to develop a set of general guidelines and recommendations that stakeholders can use to design and execute disease-specific patient preference studies. With PPMD's history designing and conducting patient preferences studies in Duchenne, we believe strongly that our…

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Added by PPMD on December 22, 2015 at 10:30am — No Comments

A New Dog Model for Drug Development in Duchenne

Photograph: Chris Kench/www.chriskenchphotography.com via The Guardian

Some…

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Added by John D. Porter, PhD on December 21, 2015 at 11:03am — No Comments


Staff
BioMarin Announces That FDA Has Advised it Will Not Take Action on Drisapersen New Drug Application by PDUFA Date

BioMarin has announced that the FDA will not make a decision regarding drisapersen until early January 2016. PPMD will continue to help both BioMarin and the FDA any way we can over the next few weeks. Thank you to the families who are spreading awareness about their experiences on drug. We will keep you updated on all developments. 

BioMarin Announces That FDA Has Advised it Will Not Take Action on the Kyndrisa™…

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Added by PPMD on December 18, 2015 at 8:30am — No Comments

Dystrophin Mutations Affect Not Only Existing Muscle Fibers, but Satellite Cells As Well

A recent paper by Michael Rudnicki’s group at Ottawa Hospital Research Institute and the University of Ottawa has provided new insights into what we view as the critical targets for therapies in Duchenne (Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division.…

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Added by John D. Porter, PhD on December 17, 2015 at 4:00pm — No Comments


Staff
PPMD Testifies at FDA PDUFA Stakeholder Meeting

PPMD's Annie Kennedy has been actively representing our Duchenne community as discussions around the next version of the Prescription Drug User Fee Act (PDUFA VI) have begun between the FDA, industry, and the patient advocacy…

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Added by PPMD on December 17, 2015 at 3:27pm — No Comments


Staff
Another matching gift for early-stage research!

We have exciting news—and even more exciting news!

We are just over halfway to our $200,000 goal—thanks to so many of you donating and the generous matching gift from the Weismans!…

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Added by PPMD on December 17, 2015 at 11:00am — No Comments


Staff
PPMD Advocates Ensure Duchenne Research Pipeline to Benefit from Congress’s “Omnibus Spending Package"

In the wee hours of this morning, Congressional leaders released the text of a massive spending bill (called the “Omnibus Spending Package”) that will fund the government for the remainder of Fiscal Year 2016, which runs through the end of September 2016. PPMD is very pleased that this bill…

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Added by PPMD on December 16, 2015 at 2:00pm — No Comments


Staff
The Drisapersen Ad Comm: Where We Are Today

We wanted to take a moment today and let you know where things stand from PPMD’s perspective since the drisapersen Advisory Committee Meeting on November 24 (…

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Added by PPMD on December 15, 2015 at 3:24pm — No Comments


Staff
Why this community matters to me

When Larry and I lost our son Peter to Duchenne more than 35 years ago, there was virtually no Duchenne community to speak of. We felt completely alone.

So much has changed in the ensuing years. To begin with, PPMD was founded. And it’s no accident that, since then, we’ve…

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Added by PPMD on December 15, 2015 at 10:25am — No Comments


Staff
Update on March 2015 Recall Notice for Deflazacort 30mg (Masters Marketing)

There has been new information regarding the March 2015 recall of Deflazacort batches JL21 and JL28 from Masters. It seems that these batches were recalled due to a suspected…

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Added by PPMD on December 14, 2015 at 1:00pm — No Comments


Staff
One Sister's Amazing #GivingTuesday Achievement

Michelle Ioannou took part in Giving Tuesday in honor of her brother Christopher, 21, who has Duchenne! To date she has raised $2,600 through her own social media and email outreach. PPMD is honored to be the…

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Added by PPMD on December 10, 2015 at 4:00pm — No Comments


Staff
PPMD Houston Roundtable: Session Recordings

In October, PPMD held a FACES Roundtable in Houston that included an incredible line up of global experts in care and research. In an effort to ensure this information reached families beyond Houston, we recorded the sessions. Special thanks to Rachel and James Poysky for pulling together such a…

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Added by Ryan Fischer on December 10, 2015 at 3:30pm — No Comments


Staff
PPMD's Pat Furlong Receives FDLI Distinguished Service and Leadership Award

For over twenty years, the Food and Drug Law Institute (FDLI) has presented the Distinguished Service and Leadership Award to individuals for their sustained…

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Added by PPMD on December 10, 2015 at 9:30am — No Comments


Staff
BIO and PPMD Launch Initiative to Share Best Practices on Patient Preference Studies

Today, the Biotechnology Industry Organization (BIO) and Parent Project Muscular Dystrophy (PPMD) announce the launch of a new initiative designed to share best practices for the development of disease-specific patient…

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Added by PPMD on December 9, 2015 at 2:08pm — No Comments

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