December 2017 Blog Posts (17)

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

Your gift helps PPMD expand gene therapy

We’ve entered a new chapter for families with Duchenne, and 2017 has been a year of incredible promise.

Yes, we’ve faced some setbacks, but we’ve also seen incredible successes, like recent breakthroughs in gene therapy. It’s essential that we keep…


Added by Abby Bronson on December 12, 2017 at 9:00am — No Comments

Santhera Update for Duchenne Community on SIDEROS Study

Santhera has provided an update to the Duchenne community, which contains updates on the SIDEROS study, educational events, respiratory guidelines/care, and more. Click here to view the update.…


Added by PPMD on December 11, 2017 at 4:30pm — No Comments

PPMD Signs Letter to Save CDC

PPMD joined 153 members of the CDC Coalition and other supporting state and national organizations in urging Congressional leaders to provide at least $7.8 billion for the Centers for Disease Control and Prevention’s programs as part of the final FY 2018 Labor, Health and Human Services, Education and Related Agencies Appropriations bill and other final FY…


Added by PPMD on December 7, 2017 at 12:20pm — 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

Duchenne/Becker and FSH Muscular Dystrophies Receive ICD-10 Codes

Parent Project Muscular Dystrophy, FSH Society Lead Effort to Obtain

Critical Diagnostic Classification Standard


Parent Project Muscular Dystrophy (PPMD)  along with collaborators the…


Added by PPMD on December 7, 2017 at 8:30am — No Comments

PPMD Designates UCSF Benioff Children's Hospital San Francisco a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named UCSF Benioff Children’s Hospital San Francisco (UCSF) our 18th Certified Duchenne Care Center.


PPMD’s Certified Duchenne Care Center Program, which…


Added by PPMD on December 6, 2017 at 9:30am — No Comments

Making Duchenne Care and Clinical Trials Better – Globally

An email from a mom in war-torn Baghdad who doesn’t know how to even start to seek care for her son with a new diagnosis.

A mom in India who can’t find a provider who will give her son steroids, but instead say stem cells will cure him.

A physical therapy student in Turkey who wants to learn all she can…


Added by Kathi Kinnett on December 5, 2017 at 11:55am — No Comments

Take Action - We Can Still Impact the Tax Reform Bill

The Senate passed their tax reform bill. In the dead of night. Overnight -- shortly before 2 AM on Saturday.

Despite protests and outcries from almost every sector: non-profits, disability communities, healthcare providers, educators, universities, researchers, graduate students (future researchers, geneticists, nurses,…


Added by Annie Kennedy on December 5, 2017 at 9:00am — 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

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