November 2014 Blog Posts (10)

what comes next

As most of you know my son Jacob has been dealing with DMD since he was 4. Well we keep getting news and all of it bad I'm just wondering when does the bad news stop and we get some good news. 

On our last visit to the doctor it went as it usually does except for the fact that he had been sick for over a month. And the doctor cant explain why so we are having to wait a month to see a specialist for his lungs because they think they are to weak to fight a common cold. He is still…


Added by danielle on November 29, 2014 at 8:56pm — 2 Comments

Finding gratitude in the bittersweet

We are just two days away from probably my favorite holiday. And I am writing a message to you, like I always do, in my little home office in Middletown before I leave town for Thanksgiving.

I am surrounded by photos of my family and normally would spend a few moments feeling nostalgic. Chris and Patrick permeate every corner of the house, and for…


Added by Pat Furlong on November 25, 2014 at 12:00pm — No Comments

PPMD and Santhera Pharmaceuticals Team Up on New Benefit/Risk Study Focused on Pulmonary Therapies

PPMD is proud to announce a collaboration with Santhera Pharmaceuticals on a benefit/risk study that will focus specifically on patient and caregiver preferences regarding pulmonary therapies in the disease, and will be based on data from Santhera’s successful phase III clinical trial of idebenone. One of the recommendations in our community’s…


Added by Holly Peay on November 25, 2014 at 9:00am — No Comments

21st Century Cures: A sign that the tide could be turning

If I were to tell you that it takes an average of 10 years and one billion dollars to yield a single new therapy – would you be surprised? Probably not, yet none of us has that luxury of time or money.

If I were to share that many of our nation’s leaders admit that our scientific and clinical institutions and the…


Added by Annie Kennedy on November 24, 2014 at 11:30am — 1 Comment

Help us bring the ABLE Act to the Floor – it’s a game changer!

Great news... House and Senate Leadership continue to hear from the disability community that the Achieving a Better Life Experience (ABLE) Act (S. 313/H.R. 647) must pass this year!

The ABLE Act amends Section 529 of the U.S. Tax Code that allows families to save for the future of their child with a…


Added by Annie Kennedy on November 21, 2014 at 9:00am — No Comments

How can we best advance the science of patient input to improve the efficiency of the drug development and regulatory review process?

PPMD and other leaders in the rare disease community were asked by PhRMA to participate in a blog conversation about how to advance the science of…


Added by Ryan Fischer on November 20, 2014 at 11:30am — No Comments

PPMD Names Baltimore’s Kennedy Krieger Institute a Certified Duchenne Care Center

Kennedy Krieger Institute in Baltimore, Maryland was named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end…


Added by PPMD on November 18, 2014 at 1:28pm — No Comments

Pfizer Myostatin Blocker Trial Set to Launch

At the Action Duchenne meeting in London on Saturday, Pfizer Inc. announced that it has completed a study in healthy volunteers with its antibody-based therapeutic to block the muscle protein myostatin, and is preparing to start recruitment for a phase II study in Duchenne this month. Watch…


Added by PPMD on November 14, 2014 at 12:59pm — No Comments

Duchenne Pulmonary Care for Tweens and Teens (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 November 6, 2014 at 10:00am — No Comments

Patients Are Waiting...

The FDA is listening and has told us as much. But that doesn’t mean we should stop doing everything we can to educate the Agency.

Thanks to the dozens of you who participated in PPMD’s benefit/risk project and took the time to share your story, we have been able to compile your responses and will be providing the FDA with…


Added by PPMD on November 3, 2014 at 7:30am — 1 Comment

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