PPMD Community

Pat Furlong's Blog (224)

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

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…


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…


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…


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…


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.…


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…


Added by Pat Furlong on March 7, 2014 at 9:30am — No Comments

Monthly Archives









© 2015   Created by PPMD.

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service