Pat Furlong's Blog (265)

Add Your Voice to the Historic Compass Meeting

There are hardly words to capture the impact our unified community had on Monday. We had traveled from all over the nation to convene with federal policymakers, industry partners, and payers for the Duchenne Patient-Focused Compass Meeting: Navigating our Pathway Forward. Nested within PPMD’s…


Added by Pat Furlong on March 8, 2018 at 2:00pm — No Comments

First Duchenne Patient Dosed in Microdystrophin Gene Therapy!

(Photo used with permission from family.)

“DMD gene therapy went well. It was started at 1:15 PM and ended at 2:27 PM”    

On Thursday, January 4, I received this message from Dr. Jerry Mendell accompanied by the photo above – Dr. Mendell at…


Added by Pat Furlong on January 10, 2018 at 10:00am — 3 Comments

How You Can Report Serious Drug Side Effects to the FDA

When our children are hurting, we go to our medicine cabinets and take great care to select products that will ease their pain. Reduce the fever. Relieve the ache.

In Duchenne, we know that the products and supplements prescribed often come with side effects. But we carefully weigh those side effects against the risk of doing nothing. We consult…


Added by Pat Furlong on January 3, 2018 at 12:00pm — No Comments

Expanding PPMD’s Gene Therapy Initiative

This holiday season, PPMD is asking you to support our ongoing Gene Therapy Initiative. Gene therapy has dominated the headlines this year in a number of diseases, including Duchenne. There is such…


Added by Pat Furlong on December 7, 2017 at 10:30am — No Comments

Invest in gene therapy--all gifts matched!

So much has changed in the Duchenne research landscape since we were founded back in 1994, but your commitment has been a constant since our story began.

You’ve never stopped fighting, and the promise for our community has never been greater, with recent breakthroughs in gene therapy, one of the most exciting approaches we’ve ever seen. Today, we…


Added by Pat Furlong on December 4, 2017 at 10:30am — No Comments

Thankful for your strength and courage

What an odd year it has been. I won’t say it has been a bad year, not entirely, but it has been dark. And I don’t think it’s just me.

Even social media, something I turn to for connection, a familiar friend, a warm embrace…has felt toxic. So many Facebook posts are gas soaked, with every “Like” a potential flame.…


Added by Pat Furlong on November 16, 2017 at 1:00pm — No Comments

SMA Gene Therapy Study: Their Success is Our Hope

Yesterday, the New England Journal of Medicine (NEJM) published “Single-Dose Gene-Replacement Therapy for Spinal Muscle Atrophy.” 

Spinal muscular atrophy (SMA) is a disorder caused by a mutation in the survival motor neuron gene 1…


Added by Pat Furlong on November 2, 2017 at 9:54pm — No Comments

The Future of Ataluren: A Hard Day But Still a Path Forward


Today the FDA issued a Complete Response Letter regarding PTC Therapeutics’ application for ataluren. Today’s news is hard. This decision is disappointing and not the outcome…


Added by Pat Furlong on October 25, 2017 at 1:22pm — No Comments

You can still join the fight to end Duchenne!

As we head into the 10th Annual Coach To Cure MD weekend tomorrow, with emotions still running high from yesterday’s…


Added by Pat Furlong on September 29, 2017 at 10:06am — No Comments

Coming Full Circle: The Ataluren Ad Comm

Like any of you who participated in today’s Advisory Committee Meeting at the FDA for PTC Therapeutic’s ataluren, I am emotionally exhausted. Whether you participated by speaking during the Open Public Hearing, attending the Ad Comm on the FDA campus, submitting your written testimony, or streaming the meeting from home – I would imagine that you, too, are…


Added by Pat Furlong on September 28, 2017 at 5:30pm — No Comments

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 now and then.


The not knowing whether you’ve…


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 ( 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 treatment for Duchenne as far back as the 1990s. If successful,…


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 community.


But today I know that there actually is something worse…


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

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