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All Blog Posts (1,803)


Staff
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…

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


Staff
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…

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


Staff
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.…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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.



Because…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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.…

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


Staff
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…

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Added by PPMD on December 7, 2017 at 12:20pm — No Comments


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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,…

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Added by Annie Kennedy on December 5, 2017 at 9:00am — No Comments


Staff
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,…

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


Staff
PPMD Research Update: Gene Therapy, Accelerating Clinical Trials & More

Throughout the history of Duchenne, there have been moments that we recognize as inflection points, times when we saw a significant shift in the fight to end Duchenne, when the…

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Added by Abby Bronson on November 30, 2017 at 6:30pm — No Comments


Staff
Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Duchenne

Solid Biosciences announced today that it has initiated clinical trial activities for SGT-001, the company’s lead microdystrophin gene transfer candidate for the treatment of Duchenne.



The Phase I/II adaptive study, called IGNITE DMD, will evaluate the safety and efficacy of a single intravenous (IV) dose of SGT-001 in ambulatory and non-ambulatory adolescents and children with Duchenne. Enrollment will begin at the first clinical trial site in the United…

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Added by PPMD on November 30, 2017 at 10:24am — No Comments

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