Pat Furlong's Blog (186)

Two weeks ago, PPMD proudly published Putting Patients First, a white paper outlining recommendations to speed responsible access to new therapies for Duchenne. In that short amount of time, the reaction to this white paper has been phenomenal. From interest by the media to accolades from industry and agencies, we have been truly humbled by the response we have received.

And while the…

April 29-30 in Washington, D.C.

Recently I had the privilege of attending and participating in the Symposium on Best Practices in Clinical Study Design of Rare Diseases in Washington, D.C. The symposium was organized in collaboration with Children’s National Medical Center (CNMC), Clinical and Translational Science Institute of Children’s National (CTSI), The George Washington University, and Research in Pediatric Developmental Pharmacology Centers (RPDP).  This…

CLINICAL TRIALS – The path to approval.

Typically we think about the Clinical Trial Process in the context of our kids.   We hear about Phase I trials.   Depending on the drug/biologic, many of these compounds will be required to be tested in healthy human subjects.   Some Phase I trials have inclusion criteria, suggesting they are looking for healthy adults of a certain age to take a single or multiple doses of a compound or biologic.   The purpose of the study…

I have met some amazingly innovative people in my travels. Many who are dedicated to improving the lives of those living with rare diseases. Wendy White is one of those people. Wendy is the founder of Siren Interactive.  Siren is a digital relationship marketing agency focused exclusively on addressing the…

PPMD is proud to share an advance copy of Putting…

Funeral dirge. Hearts break so many times, it is impossible to keep track.  A tidal wave of memories, nearly too much to bear. Minds racing, reviewing the journey that was Duchenne and all of the ‘what ifs’ and ‘if only’ that remain. Ryan’s father, mother, sisters – raw and alone, returning to the home once overflowing with Ryan and now empty.

I met Howard Kaplan many years ago in Chicago. He is a tall, broad shouldered man and in the business of protection.  His company,…

On March 26, PPMD President Pat Furlong, along with other members of the Duchenne community (internationally, including those like Pat that traveled from the U.S.) met with the UK's Shadow Minister of Health, Andrew Burnham in a meeting at Parliament.

The Video

Setting the Stage

The meeting opened with a video, that of a 4-year-old boy, Jack, diagnosed in…

When Chris and Patrick died, there was no Internet, no way to connect. The days just after they died, life was busy but at the same time heartbreaking – there was so little time to think about anything. After all was over, silence settled in. We were left with the burden of living without them, of going on and trying to make some sense of it.

No one called, not…

I wish we had a Tree of Hearts. Healthy new hearts would hang on the high branches and devices on the lower branches – low hanging fruit! Established guidelines about who, what, in what circumstances, and where, accompanied by a simple set of instructions for physicians all over the world to…

The phone rings at 6AM. Something else happens inside, a feeling that something is horribly wrong. This morning, the window of my blackberry said “BRIAN D” – Brian Denger. I imagined Brian running through the snow, nails in his sneakers to cut through the ice on the streets in Biddeford Maine.

Brian’s…

Other than Downton Abbey, TV is not my favorite sport. On occasion, I have watched CSI, The Closer, Bones, but, truth be told, they are all the same. Person murdered, investigation, fingers point one way or another, murderer confesses. End of story.  

When my boys were diagnosed with Duchenne, I fell into the same trap. Who is responsible? Both my parents smoked, so it’s their fault? My father’s company made doctor bags (Schell Leather Goods). As a child, I would ride to the…

There are no words that will make sense on a day like today. No matter the context, they will fall empty. Words are unable to grasp the enormity of losing Luke and none big enough to tell the story of his life or of your lives together. From the moment you held him in your arms…

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Photo Credit

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Every Thanksgiving, I find myself with a lot to be thankful for: my family’s love and support, our Duchenne community, and your tireless efforts.

This year is no exception. We’ve accomplished so much together. When I think of all that our community has made possible over the years, all that we’ve done in 2012, and all that we’re doing today, my heart fills with…

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So many of our friends and family, including the…

Ivy's son, Rain

What IS Duchenne?

by Pat Furlong

Last year, Ivy and I agreed to write a co-blog. I would introduce Ivy’s or wrap something…

Background 

Friends, we have been actively pursuing a regulatory strategy and have had two meetings with the neurology division at the FDA. We are currently working on the 'to do' list for the community and are trying to understand barriers/obstacles to approval from the FDA side, as well as, from the sponsor side of the issue. We are working a smart, targeted call to action. We have learned through years…

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Wow! What an unbelievable few weeks we have had in this community! Never in the 18…

This month, instead of a pair of essays, Ivy Scherbarth and I are calling for a Virtual Moment of Silence. We invite you, our Duchenne/Becker Muscular Dystrophy community, to use the comments area here to list…