Featured Blog Posts (384)

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…


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

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…


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

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 (click here…


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

A Historic Day for Duchenne

I have never been so proud of this community. Today our community reached a historic milestone, a moment I wondered if we would ever see. Today’s FDA Advisory Committee (Ad Comm) for BioMarin’s drisapersen, gave us an up close look at the regulatory process, learning firsthand what the…


Added by Pat Furlong on November 24, 2015 at 8:30pm — 2 Comments

FDA Advisory Committee Scenarios

By now, you probably know what to expect from the upcoming Advisory Committee meetings in terms of logistics: how to submit written testimony, how to apply for an open public hearing slot, order of the day, etc. (read what an Ad Comm is or …


Added by PPMD on November 17, 2015 at 1:00pm — No Comments

PPMD Supports Advancing Targeted Therapies for Rare Diseases Act of 2015 (S. 2030)

For over a year, PPMD has been working with congressional champions and industry partners to help lead an effort to streamlining the regulatory pathways and review processes for targeted therapeutics (which would include such things as 'follow on exons') by clarifying the FDA’s existing authority to leverage data previously used in the approval of a targeted…


Added by PPMD on November 12, 2015 at 1:09pm — 1 Comment

PPMD Submits Results of Patient-Centered Benefit-Risk Assessment Study in Duchenne & Becker to FDA

PPMD is pleased to announce that late yesterday we submitted the results of our most recent patient-centered benefit-risk assessment (PCBR) study to the FDA. The assessment was done with research partners at the Johns Hopkins Bloomberg School of Public Health. PCBR assessment is an increasingly-favored…


Added by PPMD on October 22, 2015 at 7:48am — No Comments

How You Can Participate in Upcoming FDA Advisory Committee Meetings




Added by PPMD on March 10, 2016 at 9:00am — No Comments

Ad Comms Update: Group Letter Submitted to FDA

PPMD is proud to help author and share the attached letter to Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research at the FDA. Along with CDMD…


Added by PPMD on October 19, 2015 at 11:00am — No Comments

PPMD Urges Senate to Move Forward on #Cures2015 Innovation for Healthier Americans Bill

Yesterday, I had the distinct privilege of representing PPMD and our Duchenne community as I sat before a full room of Senate staffers and congressional aides in the Dirksen Senate office building. Our goal was to urge them to press forward on the Innovation for Healthier Americans bill (also referred to as the ‘Innovation…


Added by Annie Kennedy on October 9, 2015 at 12:00pm — 1 Comment

New Publication Demonstrates Improved Value of Cardiac MRI in Duchenne Muscular Dystrophy

The neuromuscular group at Great Ormond Street Hospital, London UK, have recently published their study comparing the effectiveness of echocardiogram with cardiac MRI in the assessment Duchenne cardiomyopathy in patients preparing for surgical…


Added by Kathi Kinnett on September 29, 2015 at 3:00pm — No Comments

“Hullabaloo in Duchenne”

Friday was one of those days, meetings in Washington all week and anxious to get home. Traffic was a mess, Friday evening flights out of DC chaotic. I worried I would miss my flight, the last one to Cincinnati. I attempted to guesstimate the amount of time to get through the security line, grab a salad, and still make the flight. I made it just in time to learn my…


Added by Pat Furlong on September 28, 2015 at 10:00am — No Comments

ACTION ALERT: Don't Let Critical Senate Legislation Stall!

Urge your Representative to Pass the Ensuring Access to Clinical Trials Act of 2015 (H.R. 209)


One minute of your day could mean millions of minutes for those diagnosed with Duchenne.

Today, patient advocates from across the country will unite in a day of action to urge the Senate to make medical innovation a top priority.

Earlier this year the Duchenne…


Added by Ryan Fischer on September 25, 2015 at 9:30am — No Comments

FDA Advisory Committee Meetings: An Overview of the Process & Ways to be Involved

On September 2nd, PPMD convened a public webinar for the Duchenne community focused on FDA Advisory Committee Meetings (or “Ad Comms”). After working closely with representatives from FDA, we are excited to share some of the frequently asked questions that arise regarding the Ad Comm process, as well as some specifically relevant to our community. As we get closer to possible dates for these meetings, we will add information as it becomes available.…


Added by PPMD on September 24, 2015 at 3:30pm — No Comments

Webinar Recording: How FDA Ad Comms Work & What Role You Can Play

Ad Comms – What’s the buzz all about? If you’ve been anywhere near social media lately, you’ve likely seen the term ‘Ad Comm’ referenced. Speculation about dates, implications for approvals and access to therapies we all care about, questions about…


Added by Annie Kennedy on September 14, 2015 at 12:32pm — No Comments

PPMD’s DuchenneConnect to Receive $1.4 Million Funding Award from PCORI

(pictured: DuchenneConnect's Family Advisors at PPMD's 2015 Connect Conference)

, led by PPMD, has been approved for a three year, $1.4 million funding award by…


Added by Ann Lucas on August 12, 2015 at 12:04pm — No Comments

One week away from World Duchenne Awareness Day!

People around the world will help participate in the second annual World Duchenne Awareness Day on September 7th. There are a variety of ways you can get involved with this international effort – how will you raise awareness in your community?…


Added by Ryan Fischer on August 7, 2015 at 10:30am — No Comments

PPMD Submits Comments on FDA's Draft Guidance on Duchenne

We are coming to the end of the 60 day open comment period for the FDA’s draft guidance on Duchenne. PPMD submitted our comments to the agency yesterday –…


Added by PPMD on August 6, 2015 at 11:09am — No Comments

Action ALERT: Urge your Senators to Cosponsor S. 1597, the Patient-Focused Impact Assessment Act!

Call and email your Senators today! The Food and Drug Administration’s efforts to support meaningful patient engagement, also known as Patient-Focused Drug Development (PFDD), includes the strengthening of existing tools and creation of new ones needed to fill gaps, policies to allow for greater engagement of external experts in the review process, and a…


Added by PPMD on August 5, 2015 at 12:57pm — No Comments

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