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Pat Furlong's Blog (219)

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.…

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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" –…

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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…

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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…

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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process involving patients, parents, investigators, industry, and other interested parties which culminated in a draft guidance document…

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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 –…

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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…

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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…

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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…

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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…

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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…

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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…

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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…

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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.…

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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…

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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…

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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…

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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…

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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:…

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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…

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

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