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Pat Furlong's Blog (234)

Take a moment today

Dear Friends,


It has been an incredibly intense few months as we hit the homestretch of 2015 and head into a promising 2016. One Advisory Committee meeting is down and we have another quickly approaching most likely in late January 2016.…


Added by Pat Furlong on November 26, 2015 at 10:00am — 2 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…


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

Ad Comms Update: Defining Unmet Medical Need

After yesterday’s news regarding the Ad Comms for both BioMarin and Sarepta, the community continues to ask questions. PPMD has been working around the clock to get some answers…


Added by Pat Furlong on October 15, 2015 at 3:30pm — No Comments

Ad Comms Update: An Overwhelming Day

Today we learned that the FDA will host an Advisory Committee (Ad Comm) meeting on November 24 to discuss the…


Added by Pat Furlong on October 14, 2015 at 4:30pm — 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…


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FDA Releases Draft Guidance for Duchenne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 As you know, we submitted draft guidance to FDA in June,…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

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