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Pat Furlong's Blog (230)

“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…


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

FDA Releases Draft Guidance for Duchenne

Amazing things happen when this community works together, when our messages are consistent, and our goals are in sync. We work together, we change the landscape, we make history.


Today the FDA has…


Added by Pat Furlong on June 9, 2015 at 9:00am — 2 Comments

Update on the FDA Guidance

Last week we met with FDA to discuss status and potential release of guidance. As you know, we submitted draft guidance to FDA in June,…


Added by Pat Furlong on June 3, 2015 at 12:00pm — No Comments

SomaLogic Study Published: Another Piece of the Puzzle

Today, SomaLogic and PPMD - together with several other Duchenne research groups - published a significant discovery that explains a technology allowing researchers to accurately measure the individual proteins in very…


Added by Pat Furlong on May 26, 2015 at 1:30pm — 2 Comments

Saying Goodbye to Matt Petrusko, PAAC Member

Last night, another beautiful spirit in the Duchenne community left this world much too early. Matt Petrusko was a 34-year-old from Rahway, New Jersey and a proud member of PPMD’s…


Added by Pat Furlong on May 11, 2015 at 4:01pm — 1 Comment

A Light Goes Out – Goodbye to Ben Cumbo, PAAC Member

Ben Cumbo died last night. While Heaven is brighter today, our world has lost a great light.

I first met Ben in 2001. We testified about the importance of the MD-CARE Act. He was very young then, but his wisdom apparent. He talked about the cost of a Stealth bomber, on average $737 million…


Added by Pat Furlong on April 22, 2015 at 3:30pm — 4 Comments

PPMD Participating in FDA-NIH Dystrophin Methodology Meeting on Friday, March 20

John Porter and I have been invited to participate in the FDA-NIH dystrophin methodology meeting on Friday, March 20. While we said yes, we realize this is a difficult and painful topic for all of us. Dystrophin feels like the brass…


Added by Pat Furlong on March 18, 2015 at 9:30am — No Comments

BMS & The Voice of the Patient

For the last two years, global biopharmaceutical company Bristol-Myers Squibb (BMS) has organized a day when all of their drug development project teams from across the world would have the opportunity to take some time to…


Added by Pat Furlong on January 22, 2015 at 10:30am — 1 Comment

Sarepta Update: 168 Week Data

This morning Sarepta released the 168 week data on eteplirsen.  

For weeks there has been speculation, guesses, and hope about what we might learn.  We have been following the stories on social media, grateful for every photo or video. We are thrilled that the open label eteplirsen studies (confirmatory study and non ambulatory study) are actively…


Added by Pat Furlong on January 12, 2015 at 9:30am — 3 Comments

Nearly there!

We’re so close. Thanks to the strength and generosity of this incredible community, we are only $36,200 from our goal of $400,000 to fund combination therapies. There’s still time for you to help us reach our goal.…


Added by Pat Furlong on December 31, 2014 at 8:51am — No Comments

Combine therapies. Combine forces.

This year, I have been awed by the strength of our community. I think 2014 will be known as the year we effected change at every turn in clinical trials, on Capitol Hill, at the FDA, in doctors’ offices. And we did it as a community. Now it’s time to tackle the next challenge.…


Added by Pat Furlong on December 1, 2014 at 11:35am — 2 Comments

Finding gratitude in the bittersweet

We are just two days away from probably my favorite holiday. And I am writing a message to you, like I always do, in my little home office in Middletown before I leave town for Thanksgiving.

I am surrounded by photos of my family and normally would spend a few moments feeling nostalgic.…


Added by Pat Furlong on November 25, 2014 at 12:00pm — No Comments

Rebekah Cupcakesx Sands, Braveheart

Somewhere over the rainbow

Bluebirds fly

And the dreams that you dare to dream

Really do come true

Somewhere Over The Rainbow" –…


Added by Pat Furlong on September 25, 2014 at 10:04am — No Comments

The MD-CARE Act: Reflections & Projections

With the passage of the Amendments to the MD-CARE Act last week, there is unprecedented momentum for Duchenne at the federal level. And with this major victory, words and phrases echo from the…


Added by Pat Furlong on September 23, 2014 at 12:30pm — 1 Comment

MD-CARE Act Passed!

Duchenne Community Knows No Bounds

Today – September 18, 2014, our Duchenne community once again made history by demonstrating that nothing could stop us from ensuring that the life-saving research momentum of our landmark bill would be expanded to reflect the current needs of our…


Added by Pat Furlong on September 18, 2014 at 10:13pm — 2 Comments

PPMD Submits Cardiac Recommendations to FDA

As you know, PPMD recently facilitated a multi-stakeholder process involving patients, parents, investigators, industry, and other interested parties which culminated in a draft guidance document…


Added by Pat Furlong on August 18, 2014 at 1:00pm — No Comments

Duchenne in Paradise

Every year I try to escape the real world for a couple of days and retreat with my family to probably my favorite place on Earth – Dowagiac. I’ve reminisced before, but this place is magical to me. It is my childhood, it is summer, it is magic. With so much going on in our community –…


Added by Pat Furlong on August 7, 2014 at 10:30am — 1 Comment

Remembering Scott – In his own words

I had no words for Scott’s death. In fact, he had been actively dying for several years. I had hoped that he might see Rebekah for one last time, though I realized they were both too sick and living in different countries made it impossible. The good news is that many people with Duchenne never find…


Added by Pat Furlong on August 4, 2014 at 10:30am — No Comments

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

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