December 2014 Blog Posts (16)

Nearly there!

We’re so close. Thanks to the strength and generosity of this incredible community, we are only $36,200 from our goal of $400,000 to fund combination therapies. There’s still time for you to help us reach our goal.…


Added by Pat Furlong on December 31, 2014 at 8:51am — No Comments

Heart Failure Drugs Slow Deadly Damage in Duchenne, Offering New Hope

In a new study led by Dr. Subha Raman of The Ohio State University Wexner Medical Center, researchers were able to dramatically slow the rate of heart damage in patients with Duchenne muscular dystrophy by using a combination of well-established drugs.…


Added by Kathi Kinnett on December 29, 2014 at 7:00pm — 1 Comment

Now is the time for strength.

We’ve been working toward this moment for years. And the time for action is now.

When I started with PPMD in 2009, Pat Furlong and I talked a lot about what it would take to end Duchenne. We knew it wouldn’t be one thing, and we knew it wouldn’t be easy. We believed that only a combination of therapies could address the entire disease within the body.…


Added by Sharon Hesterlee on December 18, 2014 at 3:02pm — No Comments

The Solid Suit: Combining Forces to Enhance Strength

(Pictured from left to right: Stanley Nelson, MD, Center for Duchenne Muscular Dystrophy at UCLA, Thomas Sugar, PhD, Arizona State University Polytechnic School, Tom Egan, SRI International, Roy Kornbluh, SRI International, Laura Case, DPT,…


Added by Annie Kennedy on December 18, 2014 at 9:57am — 1 Comment

Short online questionnaire on Duchenne and epilepsy.

Sharing this survey from our friends at UPPMD. Please take a moment to fill it out. It is extremely short. 

News:  short online questionnaire on Duchenne and epilepsy.



Dear parents, boys and young men,  

We want to ask your attention for a scientific research project on Duchenne muscular dystrophy and epilepsy.…


Added by Ryan Fischer on December 18, 2014 at 9:52am — No Comments

Pfizer Initiates Phase 2 Study of PF-06252616 in Duchenne Muscular Dystrophy

Pfizer Inc. announced today enrollment of the first patient in a multicenter Phase II clinical trial of the investigational compound PF-06252616 in boys with Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. PF-06252616 is an experimental, infused, anti-myostatin monoclonal antibody. Myostatin is a naturally…


Added by PPMD on December 17, 2014 at 9:00am — No Comments

PPMD Names Massachusetts General Hospital a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named Massachusetts General Hospital (MGH) our sixth Certified Duchenne Care Center, recognizing the Hospital’s dedication to improving care for people living with Duchenne.

Earlier this year,…


Added by PPMD on December 16, 2014 at 2:03pm — 2 Comments

PPMD Holds Congressional Briefing

It was seven months to the day that Representatives Upton and DeGette first launched this bold initiative called 21st Century Cures. In fact, that hearing that would set in motion months and months of proposals for sweeping changes to research infrastructure and academic systems took place just across the hall from where we were now gathered. PPMD,…


Added by Annie Kennedy on December 16, 2014 at 9:30am — No Comments

Let's combine our efforts

I am Anessa Fehsenfeld, PPMD’s Board Chairman. More importantly, though, my husband Scott and I are the parents of three beautiful kids Bekah (18), Tyler (15), and Hudson (11). Tyler has Duchenne. Almost immediately after his diagnosis, I found PPMD, and they’ve been a godsend. As the mother of a teen with Duchenne, I can’t begin to tell you how much I’ve appreciated PPMD’s ability to look at the whole disease and help our family navigate his diagnosis at every age, every…


Added by PPMD on December 15, 2014 at 4:48pm — No Comments

Ventilation FAQ Booklet from the DMD Pathfinders

If you were able to listen to the third recording of the pulmonary webinar series (Adult Duchenne Pulmonary Care), you heard Dr. Sheehan mention the publication of a new booklet, “Ventilation and Duchenne” (PDF download) by the DMD…


Added by Kathi Kinnett on December 15, 2014 at 11:28am — No Comments

PPMD To Collaborate With Catabasis Pharmaceuticals, Inc. On CAT-1004 Development

Parent Project Muscular Dystrophy (PPMD) announced today a collaboration with Catabasis Pharmaceuticals, Inc. (Catabasis) to assist in a Phase 2 clinical trial of its CAT-1004 product candidate for the treatment of patients with Duchenne. As part of this collaboration, PPMD will provide funding to support participant…


Added by PPMD on December 11, 2014 at 11:23am — No Comments

PPMD Calls for Greater Transparency and Accountability of FDA Patient Engagement Efforts

Parent Project Muscular Dystrophy (PPMD) is urging Congress to build upon gains in recent years to further strengthen and enhance programs to ensure the patient voice is heeded during the drug development process during a Congressional briefing tomorrow, Tuesday, December 9th at…


Added by Ryan Fischer on December 8, 2014 at 1:30pm — No Comments

Parent Project Muscular Dystrophy Awards Akashi Therapeutics, Inc. $500,000 Grant

Parent Project Muscular Dystrophy (PPMD) has awarded Akashi Therapeutics, Inc. (Akashi) a $500,000 grant to fund clinical trials to test the safety and efficacy of an investigational new drug known as HT-100 (delayed-release…


Added by PPMD on December 8, 2014 at 10:36am — No Comments

ABLE Act Changes Disability Landscape – This just got real!

If you’re like many families, birthdays and holidays often bring gifts from friends and loved ones far and wide. As my son just celebrated his fourth birthday, he would tear through packages in the hope of finding a new Lego set or Ninja Turtle. He was a bit less interested in the envelopes that accompanied the gifts but patiently waited as Mommy read the cards to him and announced who each gift was from. And the concept of monetary gifts was a bit lost on him. The…


Added by Annie Kennedy on December 3, 2014 at 6:00pm — No Comments

Duchenne Pulmonary Care for Adults (Webinar Summary & Recording)

The lungs require the strength of many muscles to help a person to breathe and cough. As people with Duchenne grow older, the respiratory muscles, like the skeletal muscles, become weaker. Like the heart, every person with Duchenne will have pulmonary issues of some type.

The goal of pulmonary care is to allow both the prevention and management of…


Added by Kathi Kinnett on December 3, 2014 at 10:14am — No Comments

Combine therapies. Combine forces.

This year, I have been awed by the strength of our community. I think 2014 will be known as the year we effected change at every turn in clinical trials, on Capitol Hill, at the FDA, in doctors’ offices. And we did it as a community. Now it’s time to tackle the next challenge.…


Added by Pat Furlong on December 1, 2014 at 11:35am — 2 Comments

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