Registration for PPMD's 2016 Connect Conference Now Open!
This year's Connect Conference will be held in Orlando, FL from Sunday, June 26 to Wednesday, June 29, 2016. We can't wait to see you and your family there! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 1, 2016 Category: Neurology Source Type: news
PPMD Partners with AAP & CDC to Develop New Tool to Help Parents Detect Motor Development Delays
Early diagnosis and intervention have been part of PPMD ’s mission since we began and we hope a resource like this will help shorten the timeline it takes for families to receive a diagnosis. < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=c-zp1LhqqZw:tQZVeXMhA3M:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=c-zp1LhqqZw:tQZVeXMhA3M:qj6IDK7rITs" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=qj6IDK7rITs" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.o...
Source: Parent Project Muscular Dystrophy - February 24, 2016 Category: Neurology Source Type: news
PPMD Partners with AAP & CDC to Develop New Tool to Help Parents Detect Motor Development Delays
Early diagnosis and intervention have been part of PPMD’s mission since we began and we hope a resource like this will help shorten the timeline it takes for families to receive a diagnosis. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - February 24, 2016 Category: Neurology Source Type: news
ACTION ALERT: Urge House Member - Apply FDASIA Tools to Duchenne
There has never been a moment like this in our Duchenne community. Today the FDA released briefing documents on the application for eteplirsen. It is clear that we continue to face a serious regulatory challenge as a community. Urge your Representative to take action. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 18, 2016 Category: Neurology Source Type: news
End Duchenne eNews: New Year, New Hope
Catch up on the latest research and community updates in this month's End Duchenne eNews. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 15, 2016 Category: Neurology Source Type: news
A Blow to Our Community – But Our Work Continues
Today our community was dealt a painful blow as the FDA issued a Complete Response letter to BioMarin regarding the company’s New Drug Application (NDA) for Kyndrisa (drisapersen). As stated in BioMarin’s press release, “The FDA issues Complete Response letters to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form. FDA has concluded that the standard of substantial evidence of effectiveness has not been met.” (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 14, 2016 Category: Neurology Source Type: news
PPMD Responds to Revisions to “The Common Rule”
The Common Rule or the 'Federal Policy for the Protection of Human Subjects' is the policy that determines how you are consented when participating in trials and research, what types of research are considered to be ethical, and how biomedical samples acquired from patients can be used and stored. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 13, 2016 Category: Neurology Source Type: news
Dysphagia in Duchenne: practical recommendations to guide management
Dysphasia, or difficulty swallowing, effects many patients with Duchenne. Complaints of "something stuck in my throat" are not uncommon. A new article on dysphagia in Duchenne presents a clear step by step plan of care. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 8, 2016 Category: Neurology Source Type: news
Webinar 1/13/16: HOPE-Duchenne Trial - Capricor Therapeutics
Please join PPMD and Capricor as we host a webinar Wednesday, January 13 at 1pm eastern to discuss the HOPE-Duchenne trial which recently opened for enrollment in the US. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 7, 2016 Category: Neurology Source Type: news
We did it! Over $270,000 raised to support early-stage research.
Last month PPMD told you we hoped to raise $200,000 to support new, early-stage research. And how did you respond? By not only hitting our goal, but surpassing it! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 5, 2016 Category: Neurology Source Type: news
Finding Hope in CRISPR/Cas9
Duchenne made national headlines over the holidays thanks to the latest news on gene editing technology. PPMD wanted to help take a deeper dive into gene editing and CRISPR/Cas9 so that we can better understand the potential of this cutting-edge technology. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - January 4, 2016 Category: Neurology Source Type: news
Last chance to have your donation doubled!
Just a few hours left to match every single gift! We’re so close to hitting our goal—but we need to raise $24,000 more by midnight tonight. Help us take full advantage of the matching gift by making a tax-deductible donation before midnight! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 31, 2015 Category: Neurology Source Type: news
Less than 48 hours left!
We're less than 48 hours away from a New Year and just $36,000 shy of our $200,000 goal to fund early-stage research. We’re so close, but we need your gift to help us get there. Donate now—take advantage of the final hours of our match! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 30, 2015 Category: Neurology Source Type: news
Submit Written Testimony for Sarepta's Upcoming FDA Advisory Committee Meeting
PPMD encourages Duchenne community members to submit written testimonies to the FDA Advisory Committee in advance of the upcoming Sarepta Ad Comm meeting. Testimonies must be submitted by January 7th. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 28, 2015 Category: Neurology Source Type: news
" My mom and dad will match your donation! "
John Owen Dumm shares why his family and John Owen's Adventure want to match every gift made to PPMD online this holiday season. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 28, 2015 Category: Neurology Source Type: news
