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All Blog Posts Tagged 'Staff' (273)


Staff
New Duchenne Therapies in Development

As we finalize the agenda for this year’s Connect Conference, I am once again amazed at how much of a challenge it is to squeeze in all of the talks on different therapeutic approaches to treating Duchenne—in fact, there are too many to cover comprehensively this year. Sometimes it’s good to remember that mutation-specific approaches like exon skipping, although promising and…

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Added by Sharon Hesterlee on April 24, 2014 at 12:30pm — No Comments


Staff
Sarepta Therapeutics Announces Plans to Submit New Drug Application to FDA for Eteplirsen for the Treatment of Duchenne Muscular Dystrophy by Year End 2014

Great investor call from Sarepta this morning on the heels of the news about positive signals from the FDA for submitting a new drug application! We are speaking with the company this morning and will release a detailed statement this afternoon. Congratulations to everyone!







Sarepta's Press…

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Added by PPMD on April 21, 2014 at 9:30am — 1 Comment


Staff
Success on Funding Letter, Time to re-engage on the MD-CARE Act



Thanks to your efforts, we closed the House and Senate funding letters with the support of 30 House members and 12 Senators. Great work! We appreciate everyone who sent emails and made calls to their members of Congress.

The members below…

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Added by Ryan Fischer on April 15, 2014 at 9:51am — No Comments


Staff
A New Care Guide to Give to Your Provider – Imperatives for Duchenne Care

Unfortunately, the care of people living with Duchenne muscular dystrophy differs from country to country, from state to state, and even from center to center. Email and stories that we get daily confirm that, despite the availability of Care Standards there is still significant practice variability, including centers and locations where clinicians…

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Added by Kathi Kinnett on April 14, 2014 at 1:00pm — 9 Comments


Staff
PPMD Awards $100,000 in Exploratory Grants

PPMD is thrilled to award two exploratory grants as part of our ongoing grant program. Peter Arthur of the University of Western Australia was awarded $50,000 to finish testing a molecule called procysteine in animal models in preparation for a human clinical trial. Procysteine is an antioxidant that has…

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Added by Sharon Hesterlee on March 25, 2014 at 11:42am — 2 Comments


Staff
Certified Duchenne Care Center Program

Right now, there are more than 10-12,000 young people diagnosed and living in the United States with Duchenne. These young people are being cared for by clinics both near and far. Every one of them deserves the best care and treatment possible. Families want care and treatment to be transparent; they want to know who is delivering care to their child, what comprehensive care looks like, and which centers are delivering that care. For this reason, a group of experts agreed to give families…

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Added by Kathi Kinnett on March 20, 2014 at 9:00am — 2 Comments


Staff
3 Actions You Can Take to Tell the FDA to Speed the Approval of New Therapies for Duchenne

As a community, we must continue to demand that the FDA use all available tools given to them by Congress to speed the approval of new therapies to all who so desperately need them. The voices of patients and caregivers are critical to the process of drug development and we believe the message is being heard.

 

Here are 3 simple actions you can take to support & amplify that message:

 

  1. Urge your…
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Added by Ryan Fischer on March 19, 2014 at 12:10pm — No Comments


Staff
Simple Actions: Real data can drive change

For years our community has been making noise so that the scientific and medical communities would listen and focus on Duchenne. They're listening – now we need to talk! Filling out a survey or doing an interview about your family's experiences with Duchenne creates important data. This data…

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Added by Holly Peay on March 18, 2014 at 9:30am — No Comments


Staff
Prosensa Announces Drisapersen 48-Week Data and Recent Corporate Developments

Prosensa just released their 48-week data for drisapersen, as well as an update on recent corporate developments. Both press releases are included below.



PPMD has been talking with Prosensa and will be hosting a webinar with UPPMD…

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Added by PPMD on March 18, 2014 at 9:00am — No Comments


Staff
Action Alert- Ensuring Federal Agencies Working on Duchenne Receive Proper Funding

Though the MD-CARE Act remains our top priority, we need to ensure that agencies receive adequate funding and direction for the programs that are so critical to our community. One tool we use to ensure that funding and direction is our Congressional funding letter. It has made a tremendous impact on appropriations. 

To illustrate this point, let’s look at how our yearly funding letters have impacted the NIH’s…

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Added by Ryan Fischer on March 14, 2014 at 9:30am — 4 Comments


Staff
In Support of Approvals

As a mother who lost two sons to this dreadful disease, I am driven to find every way possible to help bring new therapies to families, to end Duchenne. PPMD supports and agrees with all parents urging the agency to accept Sarepta’s submission for the approval of eteplirsen, as well as any other therapy that shows similar promise. The voices of patients and caregivers are critical to the process of drug development and we believe the message is being heard.



PPMD is…

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Added by Pat Furlong on March 7, 2014 at 9:30am — No Comments


Staff
Contemporary Cardiac Issues in Duchenne Muscular Dystrophy

We all know that the heart is a muscle too! Most people and parents living with Duchenne feel that care for the heart, as defined by the Care Considerations, is not as current or as aggressive as they would like. In 2009, when the Care Considerations were published, cardiac care was very…

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Added by Kathi Kinnett on March 5, 2014 at 1:30pm — No Comments


Staff
MD-CARE Act: Adding New Cosponsors

It's been just one week since the 2014 Advocacy Conference, and we have already upped the number of cosponsors for the MD-CARE Act! We are now at 85 in the House & 21 in the Senate. Please take a moment to reach out to our newest cosponsors…

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Added by Ryan Fischer on March 4, 2014 at 4:00pm — 1 Comment


Staff
Nutrition, Energy, and Duchenne

During my years of clinical practice, I was repeatedly asked, “Is there something that I can feed my child to help slow down, or stop, the effects of Duchenne?” The absolute answer is – we don’t know. But a new article out this week has endeavored to address this question.



As people…

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Added by Kathi Kinnett on February 26, 2014 at 12:00pm — 2 Comments


Staff
Finally, Some Good News!

Sarepta’s 120 week stability data on both the six minute walk test, as well as, pulmonary function is good news for the Duchenne community. For the 12 young men in the study, we are thrilled. And for those waiting, we see hope on the horizon. We congratulate Sarepta for their continued commitment to people with Duchenne and we hope the FDA…

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Added by Pat Furlong on February 6, 2014 at 1:30pm — No Comments


Staff
FDA Guidance Update: Representing the Duchenne Community

An update on developing "Draft Guidance on the Clinical Investigation of drugs and biologics for Duchenne and Becker muscular dystrophy"



 

One of PPMD’s priorities is speeding development of reliable diagnostics and safe and effective…

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Added by Ryan Fischer on January 28, 2014 at 2:00pm — No Comments


Staff
Imaging DMD

Patrick was 8 years old. Steroids were not recommended. There was no imaging and no way to predict his loss of ambulation. Sure, he fell occasionally. Some days more than others. There was one day in the summer of 1988. We had been active most of the day. We went swimming, to the mall, and ended the day with a cookout. Patrick walked and walked. He asked me to help him to the bathroom. He fell. It was one of those FALLS, as if his legs were pulled out from under him. I had no excuses in my…

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Added by Pat Furlong on January 23, 2014 at 9:00am — No Comments


Staff
Prosensa Patient Webinar - January 21

Prosensa will organize a patient focused webinar on Tuesday, January 21st at 8 AM ET / 14hrs CET. Practical details required to dial in for this webinar are provided on the Prosensa website at:  http://ir.prosensa.eu/events.cfm (open additional information by clicking on it).

If you have any questions that you would like to see answered during the webinar, these can be…

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Added by PPMD on January 20, 2014 at 9:31am — No Comments


Staff
One Simple Action: Running for those who can’t

A family of 11…9 kids (the youngest with Duchenne), 2 parents and ALL of them participated in last weekend’s Walt Disney World Marathon. Bob McDonald, a PPMD Board Member, and each member of his family ran in at least one of the Disney races.  Even his youngest son Mark, with Duchenne, participated via wheelchair. Everyone in the family took a little time and effort (okay, maybe a lot!) and did something to help end Duchenne.…



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Added by Will Nolan on January 16, 2014 at 10:51am — No Comments


Staff
Register for the 2014 Advocacy Conference (Feb 23-25)

You are invited to join us in Washington (February 23-25) for what is going to be another critical year in Duchenne advocacy. As you know our efforts last year were focused on collecting cosponsors for the MD-CARE Act (MDCA) reauthorization. To date we stand at …

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Added by Ryan Fischer on January 16, 2014 at 10:00am — No Comments

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