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Pat Furlong's Blog (243)

September 24: Every Single Coach

Last week you heard from my friend Kim Powers. She and her husband have jumped into this year’s …


Added by Pat Furlong on August 10, 2016 at 11:30am — No Comments

PTC Therapeutics Provides Regulatory Update on Translarna™

PTC recently participated in discussions with FDA to discuss the Refuse to File (RTF) letter issued on February 22, 2016 with respect to the company's New Drug Application (NDA) for Translarna for the treatment of Duchenne. Today PTC…


Added by Pat Furlong on July 25, 2016 at 12:00pm — No Comments

BioMarin Discontinues Clinical Development of Drisapersen

Today is June 1 and I know many of us feel like 2016 has beat up this community already. With every small victory, we face another major setback.


As you have heard by now,…


Added by Pat Furlong on June 1, 2016 at 9:57am — 2 Comments

Every single carrier.

I am a genetic carrier of Duchenne. My mother was not. This means I was one of the 1:4600 females born with this genetic mutation. As a child, I had muscle cramps, ‘growing pains’ my parents would say. And from those ‘growing pains,’ 20 years later, my sons were diagnosed and I first learned…


Added by Pat Furlong on May 9, 2016 at 10:07am — 4 Comments

Changing the Paradigm: The Eteplirsen Ad Comm

There is no way around it, today was hard. Hundreds of us – the largest gathering of Duchenne families in history – came together to tell the FDA that eteplirsen is safe, effective, and should be approved using the tools…


Added by Pat Furlong on April 25, 2016 at 10:30pm — 5 Comments

Growing Our Team

We are not a team because we work together. We are a team because we respect, trust, and care for each other. – Vala Afshar


I am the first person to admit that I don’t know much about a lot of different things. But I do know talent when I see it. And I could not be…


Added by Pat Furlong on February 1, 2016 at 9:43am — No Comments

A Blow to Our Community – But Our Work Continues

Today our community was dealt a painful blow as the FDA issued a Complete Response letter to BioMarin regarding the company’s New Drug Application (NDA) for Kyndrisa (drisapersen). As stated in BioMarin’s press release, “The FDA issues Complete Response letters to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form.  FDA…


Added by Pat Furlong on January 14, 2016 at 12:30pm — 3 Comments

We did it! Over $270,000 raised to support early-stage research.

Last month we told you we hoped to raise $200,000 to support new, early-stage research. Throughout the month, we told you about potential therapies like epicatechin. We told you about generous matches from Mary-Lou and Larry Weisman and the Dumm Family/…


Added by Pat Furlong on January 5, 2016 at 11:00am — No Comments

Every gift doubled--for every single family.

Dear Friends,


This community should be proud of the role we have played over the last several weeks preparing for the potential approval of multiple therapies to treat Duchenne. As I mentioned last week, there is still…


Added by Pat Furlong on November 30, 2015 at 3:00pm — No Comments

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

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