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Pat Furlong's Blog (255)

Celebrate Dad & Your Gift is Doubled!

This Father’s Day, I hope you will join me in celebrating the dads in this community. They are often the unsung heroes of our community. They are role models for our sons, providing for our families. Their hearts ache just as profoundly when our children are hurting. Their hearts love just as unconditionally.

That’s why this Father’s Day, I…


Added by Pat Furlong on June 14, 2017 at 12:30pm — No Comments

Deciphering What We Know About EMFLAZA

The unknowns. Some of the hardest beasts to conquer in life.


The unpredictable variables that make life impossible to plan.


The not being able to commit to vacations and holiday plans because – well, you just don’t know what curve balls life will toss at you between…


Added by Pat Furlong on May 15, 2017 at 9:30am — 1 Comment

Deflazacort Approved in the U.S. for Duchenne

UPDATE 2/10: Marathon has released links to new resources, including the EMFLAZA website (https://emflaza.com) and a Patient Support FAQ document (…


Added by Pat Furlong on February 9, 2017 at 5:00pm — 2 Comments

PPMD Awards Nationwide Children’s Hospital $2.2 Million Grant to Explore Gene Therapy in Duchenne

Today, PPMD announced a $2.2 million dollar grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital.  Our purpose in awarding this grant is to open and accelerate the field of gene therapy – a strategy that seemed impossible as a potential…


Added by Pat Furlong on January 6, 2017 at 9:00am — No Comments

Two simple words...THANK YOU!

We not only met our goal, we surpassed it!

We could not be more overwhelmed by the incredible response to our holiday campaign. In many ways, with a therapeutic pipeline full of promising treatments, it feels like the future of Duchenne therapy is here. Thanks to this community, we head into 2017 with…


Added by Pat Furlong on January 4, 2017 at 3:47pm — No Comments

Your gift to CRISPR will be Matched!

We have so much momentum. Let’s seize this moment.

First of all, let me say THANK YOU! Your gift to PPMD on #GivingTuesday helped us surpass our goal, and we could not be more grateful. It is a wonderful way to kick off our holiday season—and we wanted to make sure you were among the…


Added by Pat Furlong on December 1, 2016 at 9:30am — 1 Comment

A truly thankful moment


Added by Pat Furlong on November 23, 2016 at 5:30pm — No Comments

The Latest Update on Translarna from PTC Therapeutics

The journey has been long. From PTC Therapeutics' first presentation about targeting a ‘stop sign’ within the dystrophin mutation to PTC 124 to ataluren and now, Translarna. The journey has been a long one.

The team at PTC are pioneers, the company…


Added by Pat Furlong on October 26, 2016 at 12:30pm — 1 Comment

PPMD Demands Anthem Reconsider Coverage of Approved Drug

It is hard to imagine anything worse than receiving the diagnosis of Duchenne. The dates and odysseys that brought us into this community differ, but the fervor we share to slow and stop the progression of this disease as it marches through our children is unparalleled in any…


Added by Pat Furlong on October 14, 2016 at 12:02pm — No Comments

FDA Grants Accelerated Approval to First Drug for Duchenne Muscular Dystrophy

Today, my sons are dancing! Today we ALL celebrate the first FDA approval in Duchenne. Today the FDA approved Exondys 51 (aka eteplirsen) injection, specifically indicated for patients…


Added by Pat Furlong on September 19, 2016 at 1:30pm — 4 Comments

September 7: World Duchenne Awareness Day

For the third year in a row, the global Duchenne community will come together on September 7 for World Duchenne Awareness Day. This day, this simple idea, has blossomed into a truly incredible opportunity for families and friends…


Added by Pat Furlong on August 31, 2016 at 2:30pm — No Comments

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

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