All Blog Posts Tagged 'PPMD Blog' (819)

Action Alert! Americans with Disabilities Act (ADA) in Jeopardy

 Despite bold outcry from disability and civil rights groups, HR 620 has passed in the House. PPMD has been in touch with Senate leadership and the path in the Senate is uncertain, but we urge our community to…


Added by Annie Kennedy on February 12, 2018 at 9:30am — No Comments

From our Hearts to Yours — Updates in Duchenne Cardiac Care Guidelines

Care for the heart has long been a priority for PPMD. Over the past several years, PPMD’s Cardiac Initiative has given rise to:


Added by Kathi Kinnett on February 7, 2018 at 1:31pm — No Comments

Summit Therapeutics Shares Interim 24-Week Data from PhaseOut DMD Clinical Trial

Today, Summit announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid.…


Added by PPMD on January 25, 2018 at 10:00am — No Comments

Updated Care Consideration Guidelines for Duchenne Published

PPMD is thrilled to announce that after many years in development, the Centers for Disease Control (CDC) has published updated Care Considerations for Duchenne in The Lancet Neurology. These articles are also now…


Added by Kathi Kinnett on January 24, 2018 at 10:30am — No Comments

PPMD's 2018 Advocacy Conference | Schedule of Events

The PPMD Advocacy Conference registration is live! Join us March 4-6 in Washington DC to advocate for Duchenne directly with Members of Congress.



Added by Ryan Fischer on January 23, 2018 at 4:50pm — No Comments

Meet the 2018 PPMD Adult Advisory Committee (PAAC)

In January of 2014 we held our inaugural PAAC Leadership Workshop in Washington,DC. The day included engagement with federal policy makers, congressional staff, and research leaders. At the end of the day, a long-time colleague of mine from the FDA who had spent the entire day with us pulled me aside and said, “Wow. These men are brilliant. They have such…


Added by Annie Kennedy on January 17, 2018 at 10:30am — 2 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

2018 Off to a Strong Start — 'Patient Experience Data’ Section Added to FDA Reviews

Remember that hot June day in 2015 when hundreds of Duchenne community members rallied together at the U.S. Capitol for PPMD’s EndDuchenne rally? That same day, our longtime Congressional champions Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) introduced the Patient Focused Impact Assessment Act (PFIA, S. 1597) — and our 200+ PPMD advocates…


Added by Annie Kennedy on January 9, 2018 at 3:30pm — No Comments

PPMD's Certified Duchenne Care Center Program – 2017 Impact & Progress

PPMD's Certified Duchenne Care Center (CDCC) Program was developed to make comprehensive Duchenne care and services, provided in agreement with the…


Added by Kathi Kinnett on January 8, 2018 at 4:30pm — No 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

We did it! Because of your commitment & courage!

Parent Project Muscular Dystrophy believes in the promise that gene therapy holds for the Duchenne community. And so we set an ambitious fundraising goal for our Gene Therapy Initiative. In fact, $500,000 is our largest fundraising goal ever.

And once…


Added by PPMD on January 3, 2018 at 9:30am — No Comments

Two days left to reach our goal

I am a 22 year old living with Duchenne, and opportunities for clinical trial participation are few and far between. It is one of the frustrating realities of my diagnosis and my age. Although it has never swayed my commitment to this community, I have to be honest. It can be discouraging at…


Added by PPMD on December 30, 2017 at 10:03am — No Comments

Great News: Match extended by PPMD board!

I have some exciting news: Your response to Sean and Mindy Rice’s generous matching gift of $200,000 has been incredibly inspiring to the Parent Project Muscular Dystrophy board. You’ve shown us that PPMD’s Gene Therapy Initiative matters to you, and we want to keep up the momentum.…


Added by PPMD on December 26, 2017 at 3:00pm — No Comments

Gene therapy gives me the courage to hope

My son Bazi is four years old and has Duchenne muscular dystrophy. It's been over a year now, and this sentence is still extraordinarily hard for me to write.

When we got the diagnosis, my emotions shifted between paralyzing fear, incredible…


Added by PPMD on December 22, 2017 at 11:30am — No Comments

What You Need to Know about Duchenne & Viral Gastroenteritis (That Horrible Stomach Bug!)

Viral gastrointestinal (GI) viruses are no fun for anyone, but they are especially worrisome for a person living with Duchenne muscular dystrophy. GI viruses affect the GI track – the stomach and intestine (i.e., ‘gut’) – resulting in abdominal pain/discomfort, nausea, vomiting, intestinal pain/discomfort, cramps and diarrhea. Thank goodness they usually have…


Added by Kathi Kinnett on December 21, 2017 at 10:30am — No Comments

Understanding Currently Recruiting Gene Therapy Studies

For the last many months, we have all been watching gene therapy make headlines around the world, across various diseases. As you know, earlier this year PPMD launched a Gene Therapy Initiative to help support the exploration of various gene therapy technologies in Duchenne.

Of particular note has been the progress made in…


Added by Ann Martin on December 19, 2017 at 4:19pm — 1 Comment

We're doubling your donation for gene therapy


When our son, Jonathan, was diagnosed with Duchenne, we didn’t know where our family would find the strength to fight. But the PPMD community has always shown us that we are never alone in our fight to end Duchenne.



Added by PPMD on December 19, 2017 at 2:56pm — No Comments

Understanding Gene Therapy: The Screening Process

With advancements made in Duchenne gene therapies now coming to fruition, a number of questions are being asked about who may qualify for the upcoming trials.


While the inclusion criteria of each trial will be different based on age, mutation (in some cases), and functional abilities, one of the criteria for participating in…


Added by PPMD on December 13, 2017 at 4:00pm — 1 Comment

PTC Therapeutics Shares Update on Ataluren Formal Dispute Resolution Request Process

PTC Therapeutics provided a brief update on the Formal Dispute Resolution Request process for ataluren.

Read the update from PTC:

Dear Duchenne Community,

As we enter the holiday season, I wanted to thank the community for all your support throughout the regulatory…


Added by PPMD on December 13, 2017 at 11:00am — No Comments

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