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All Blog Posts Tagged 'Staff' (362)


Staff
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.…



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


Staff
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…

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Added by Annie Kennedy on December 18, 2014 at 9:57am — No Comments


Staff
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,…

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


Staff
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…

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Added by PPMD on December 16, 2014 at 2:03pm — No Comments


Staff
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…

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


Staff
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,…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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.…

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


Staff
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.…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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Added by Kathi Kinnett on November 6, 2014 at 10:00am — No Comments

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