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January 2014 Blog Posts (11)


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An update on developing "Draft Guidance on the Clinical Investigation of drugs and biologics for Duchenne and Becker muscular dystrophy"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

how old were your kids when put in a wheelchair

I know i have heard from a few parents that say there kids were not reliant on a chair until like age 13-16. Well my son is 8 and he has lost almost all hip muscles and is now using a manual chair and a walker. When using the walker he is still falling and sometimes he loses his balance and falls out of his chair as well. Im just curious as to how old your kids are in chairs and what u did to stop the progression.

Added by danielle on January 22, 2014 at 10:49pm — 3 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
Prosensa Looks To Take DMD Programs Forward; Remains Hopeful For Drisapersen

Prosensa Looks To Take DMD Programs Forward; Remains Hopeful For Drisapersen

ByJoseph Haas / Email the Author / View Full Issue

Word Count: 963 /…

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Added by PPMD on January 16, 2014 at 10:35am — 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


Staff
Avoiding the Emergency Room

No one wants to be sick or go to the Emergency Room unless they absolutely need to. Keeping your lungs clean and your breathing easy will help you to avoid the Emergency Room. Unfortunately, winter is the time for respiratory illnesses and colds. During a respiratory illness/cold, your weak cough can become…

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Added by Kathi Kinnett on January 16, 2014 at 9:00am — 1 Comment


Staff
Superheroes Descend on Disney to End Duchenne



“We will fight for our sons, we will unite for our sons, and yes, we will run for our sons.”



With those words, Mike Gaglianone, our parent speaker at our team pasta dinner, kicked off an incredible weekend at the Walt Disney World Marathon Weekend January…

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Added by Nicole Herring on January 15, 2014 at 3:30pm — No Comments

FDA Public Workshop Report – Our Message Is Having an Impact

Last week, Pat Furlong and I were invited by the FDA to their meeting on Complex Issues in Developing Drugs and Biological Products for Rare Diseases. I was asked to participate on the Tolerating Risk and Uncertainty in Pediatric Clinical Trials Panel, in recognition of the work PPMD has done…

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Added by Holly Peay on January 15, 2014 at 11:00am — No Comments


Staff
College Course Offered in Duchenne

A course on Duchenne is being offered by the Ohio State University College of Medicine and Nationwide Children's Hospital in Columbus and is open to the public via…

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Added by PPMD on January 8, 2014 at 12:00pm — 5 Comments

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