PPMD Advocates Ensure Congress Recognizes Importance of Flexibility in FDA Reviews
Great news as Congress continues to communicate urgency to the FDA to apply flexibility in review of Duchenne therapies, thanks to our PPMD community! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 19, 2016 Category: Neurology Source Type: news
PPMD Names Children ’s Hospital Los Angeles 10th Certified Duchenne Care Center
PPMD is thrilled to announce our 10th Certified Duchenne Care Center -- Children's Hospital Los Angeles! < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=V5dA-mjqpBg:apUgVWfvvX4:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=V5dA-mjqpBg:apUgVWfvvX4:qj6IDK7rITs" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=qj6IDK7rITs" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=V5dA-mjqpBg:apUgVWfvvX4:V_sGLiPBpWU" > < img src="http://feeds.feedburner.c...
Source: Parent Project Muscular Dystrophy - April 19, 2016 Category: Neurology Source Type: news
PPMD Names Children’s Hospital Los Angeles 10th Certified Duchenne Care Center
PPMD is thrilled to announce our 10th Certified Duchenne Care Center -- Children's Hospital Los Angeles! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 19, 2016 Category: Neurology Source Type: news
PPMD's Every Single [One] Tour Launched in Baltimore!
On Saturday, Duchenne community members from six states gathered together at Kennedy Krieger in Baltimore to hear the latest developments on clinical trials, clinical care practices, carrier programs, legislative and regulatory efforts, IEPs and school advocacy, physical therapy, genetic testing and more. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 18, 2016 Category: Neurology Source Type: news
Senate Sends Strong Letter to FDA about Duchenne Therapies
With this letter now sent, both the House & Senate have communicated a strong message to the FDA about the need to use every possible tool at their disposal to speed access to promising Duchenne therapies. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 18, 2016 Category: Neurology Source Type: news
PPMD ’s Patient Preference Study about Pulmonary Outcomes – What we learned and why it matters
We are pleased to release the Community Report for this study, which takes a deeper look into the results. Later this year, an extended version of the report data will be published in multiple publications. < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=v9Bw5qir05c:x5NRyonek3E:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=v9Bw5qir05c:x5NRyonek3E:qj6IDK7rITs" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=qj6IDK7rITs" border="0" > < /img > < /a > < a href="http://feeds.pare...
Source: Parent Project Muscular Dystrophy - April 13, 2016 Category: Neurology Source Type: news
PPMD’s Patient Preference Study about Pulmonary Outcomes – What we learned and why it matters
We are pleased to release the Community Report for this study, which takes a deeper look into the results. Later this year, an extended version of the report data will be published in multiple publications. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 13, 2016 Category: Neurology Source Type: news
PPMD's 2016 Connect Conference Agenda Now Available
PPMD is excited to unveil the agenda for the 22nd Annual Connect Conference in Orlando, June 26-29. Make sure you register you and your family before the Early Bird Registration Rate ends April 15th! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 11, 2016 Category: Neurology Source Type: news
ACTION ALERT: Please urge Your Senators to Sign Letter to FDA
Please urge your Senators to sign this important letter. Patients need and deserve therapeutic options and the FDA must use the tools they have been given to expedite these options. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 5, 2016 Category: Neurology Source Type: news
How You Can Help with the Upcoming Ad Comm for Eteplirsen
We are just a few short weeks from the FDA Advisory Committee Meeting for eteplirsen on April 25th. The momentum is tremendous but we need YOU to take action leading up to the big day. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 4, 2016 Category: Neurology Source Type: news
Webinar Recording: BMS Anti-Myostatin Adnectin Program
PPMD and Bristol-Myers Squibb (BMS) recently hosted a webinar to discuss the BMS Anti-Myostatin Adnectin Program. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 1, 2016 Category: Neurology Source Type: news
PPMD Community Survey about the Akashi HT-100 study
If you or your child were in the Akashi HT-100 clinical trial that was recently suspended, please take our survey. Responses are needed by April 10th. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 1, 2016 Category: Neurology Source Type: news
Update on Gene Replacement/Repair Strategies for Duchenne Muscular Dystrophy
There has been a flurry of press recently on gene therapy in Duchenne and CRISPR technology. With this barrage of information, PPMD wanted to take a deeper dive into gene therapy as a potential treatment for Duchenne. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 25, 2016 Category: Neurology Source Type: news
PPMD is Initial Partner in OnPAR
PPMD is proud to participate in OnPar which provides access to peer-reviewed research proposals that directly relate to Duchenne research. OnPAR will provide us with an efficient way to solicit early stage and translational Duchenne research proposals, see the breadth of research in Duchenne, and gain access to novel research strategies. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 24, 2016 Category: Neurology Source Type: news
PPMD Launches the Every Single [One] Tour
For 22 years, PPMD has brought together families with leading experts, care providers, and industry partners through our Annual Connect Conference, FACES meetings, and Roundtables. Now, in an effort to reach every single family facing a Duchenne diagnosis in the U.S., PPMD is launching a multi-year community experience called the Every Single [One] Tour. Combining each of the pillars that make up PPMD’s mission – To End Duchenne – the Every Single [One] Tour will bring updates on research, advocacy, and care to half a dozen cities in 2016. We hope you can join us! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 4, 2016 Category: Neurology Source Type: news
