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Pat Furlong's Blog (212)

BREAKING NEWS: Houses of Representatives Passes MD-CARE Act Amendments!

The House of Representatives today approved the landmark MD-CARE Act so the law can continue getting results for all patients impacted by Duchenne and other forms of muscular dystrophy. The House approved the bill, H.R. 594, sponsored by Congressman Dr. Michael Burgess of Texas and Eliot Engel of New…


Added by Pat Furlong on July 28, 2014 at 6:12pm — No Comments

Conference 20

If you are always trying to be normal, you will never know how amazing you can be.

― Maya Angelou

What do you want to be when you grow up? The question Dr. Sue Apkon asks every boy with Duchenne (girls too). The question, that for so many years brought tears to our eyes and a lump in our throat. The question that always resulted in mental math... if this happens and that happens, then maybe…. But now, it is reality. Our sons…


Added by Pat Furlong on July 2, 2014 at 11:00am — No Comments

PPMD Welcomes John Porter!

John Porter, PhD is a Program Director at the NIH/NINDS, responsible for managing a portfolio of research grants across neuromuscular disorders. That is his formal introduction, but the other side is that he has long been a friend to me and to our Duchenne community. In January, he announced his intention to…


Added by Pat Furlong on June 28, 2014 at 5:43pm — No Comments

Strength Happens Together: PPMD Submits FDA Draft Guidance on Duchenne

June, 25, 2014 – Today is a landmark day for PPMD and the entire Duchenne community. We are so pleased that at our organization’s 20 year anniversary – after 2 decades of hard work and resolute persistence within the policy, legislative and regulatory arena – we are today able to provide to the US Food and Drug…


Added by Pat Furlong on June 25, 2014 at 11:00am — No Comments

Congressional Committee That Funds the FDA Recognizes PPMD-Led Efforts on Benefit/Risk, Draft Guidance; Applauds Collaboration with Agency

For the first time ever, the appropriations bill that funds the Food and Drug Administration includes report language focused on Duchenne muscular dystrophy and championed by PPMD advocates.


This week the House Appropriations Committee approved the Fiscal Year 2015 Department of Agriculture and FDA…


Added by Pat Furlong on May 30, 2014 at 11:22am — No Comments

A Good Day for Duchenne

EMA grants Conditional Approval for Ataluren


Ataluren, PTC Therapeutics’ nonsense suppression drug received Conditional…


Added by Pat Furlong on May 23, 2014 at 9:30am — 6 Comments

At Long Last, A Victory!

One Life on this earth is all that we get, whether it is enough or not enough, and the obvious conclusion would seem to be that at the very least we are fools if we do not live it as fully and bravely and beautifully as we can.…


Added by Pat Furlong on April 21, 2014 at 6:01pm — 1 Comment

In Support of Approvals

As a mother who lost two sons to this dreadful disease, I am driven to find every way possible to help bring new therapies to families, to end Duchenne. PPMD supports and agrees with all parents urging the agency to accept Sarepta’s submission for the approval of eteplirsen, as well as any other therapy that shows similar promise. The voices of patients and caregivers are critical to the process of drug development and we believe the message is being heard.

PPMD is…


Added by Pat Furlong on March 7, 2014 at 9:30am — No Comments

Finally, Some Good News!

Sarepta’s 120 week stability data on both the six minute walk test, as well as, pulmonary function is good news for the Duchenne community. For the 12 young men in the study, we are thrilled. And for those waiting, we see hope on the horizon. We congratulate Sarepta for their continued commitment to people with Duchenne and we hope the FDA…


Added by Pat Furlong on February 6, 2014 at 1:30pm — No Comments

Imaging DMD

Patrick was 8 years old. Steroids were not recommended. There was no imaging and no way to predict his loss of ambulation. Sure, he fell occasionally. Some days more than others. There was one day in the summer of 1988. We had been active most of the day. We went swimming, to the mall, and ended the day with a cookout. Patrick walked and walked. He asked me to help him to the bathroom. He fell. It was one of those FALLS, as if his legs were pulled out from under him. I had no excuses in my…


Added by Pat Furlong on January 23, 2014 at 9:00am — No Comments

Yesterday's Duchenne Policy Forum

Silver Springs, Maryland

12/12/13  8:30 AM 

There are days when the stars align just right. Seems to me, December 12, 2013 was one of those days.

Eighteen members of the FDA arrived and were seated around a U-shaped table near the front of the…


Added by Pat Furlong on December 13, 2013 at 12:30pm — 1 Comment

Move Duchenne research forward faster with one simple action

Dear Friends,

For our families, Duchenne research can’t move fast enough.

There is no lack of ideas or interest—7 public companies and more than 15 biotechs are focused on finding treatments for Duchenne. But promise is only one part of the story. We need to actually change the landscape and bring drugs to market faster.

You can help make it happen with one simple action:…


Added by Pat Furlong on December 4, 2013 at 11:00am — No Comments

Grateful for you

2013 has been a year of incredibly high highs and incredibly low lows. Sometimes those lows have overshadowed all the highs—and for good reason. It’s impossible to put into words how devastating the setbacks are.

But our community is and has always been stronger than the setbacks. We may be fighting a disease that takes away muscle, but you are the strongest people I’ve ever known. It’s during our biggest challenges that strength matters most, and I know we’ll get through this…


Added by Pat Furlong on November 26, 2013 at 1:30pm — 5 Comments

Time to Unite & Rally

Yesterday was a disappointing day for all parents, grandparents, aunts, uncles, and others touched by Duchenne. Like most of our community, I awoke to the news that multiple factors have led Sarepta to announce that it would delay the filing of its much-anticipated…


Added by Pat Furlong on November 13, 2013 at 12:30pm — 7 Comments

Share Your Story with the FDA

PPMD is proud to host a Policy Forum on clinical trials of experiment agents on December 12, 2013 from 8.30AM until 4.30PM in Silver Springs, MD. We are especially pleased that key Duchenne-community…


Added by Pat Furlong on November 6, 2013 at 3:00pm — No Comments

10/14 – 10/18: An Amazing Week in Review

When Chris and Patrick were diagnosed, there was no genetic testing. No therapies were targeted to the “CODE” and no way to DECODE Duchenne. We made assumptions about carrier status and lived many years with only assumptions. I thought my mother was a carrier. I knew I was a carrier. The doctor made that…


Added by Pat Furlong on October 23, 2013 at 10:30am — No Comments

How to Decode Duchenne

We are excited to announce that Parent Project Muscular Dystrophy and Sarepta Therapeutics are joining forces on a genetic testing program for patients with Duchenne or Becker…


Added by Pat Furlong on October 17, 2013 at 9:00am — No Comments

Demystifying the FDA Meeting, September 10, 2013

For many years, patient representatives have been invited to participate in meetings around drug development as the patient voice is extremely important if reviews are to understand how patients weigh benefit and risk within the context of their disease. I was honored to present earlier this week at meetings for NINDS and the FDA. Below are some of the notes I took and some of the information I heard that I wanted to share with you all.


How does it work?  FDA issues guidance…


Added by Pat Furlong on September 13, 2013 at 2:21pm — No Comments

Sarepta Therapeutics announces plans to submit New Drug Application to FDA for Eteplirsen – A significant step for the Duchenne community

Today is a landmark day for the entire Duchenne community. Not too long ago, the Duchenne therapy landscape was a barren field with little to no life. Thanks to a commitment to our patients and the field by the U.S. government, international governments, the venture community and industry, this landscape has changed markedly to the point where today we have the announcement for a…


Added by Pat Furlong on July 24, 2013 at 11:00am — 1 Comment

7.9.13: Update from PPMD/FDA Meeting

July 9, 2013.  

PPMD met with Dr. Janet Woodcock and leadership from the Office of New Drugs, Medical Policy and the Division of Neurology Products, as well as, staff from the Office of Strategic Programs, Office of Health and Constituent Affairs.   The purpose of the meeting was to continue our efforts to provide the agency relevant information and data about Duchenne that will encourage/enable them to be flexible in their review process.  



Added by Pat Furlong on July 12, 2013 at 10:39am — No Comments

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