" Why this community matters to me "
Mary-Lou Weisman reflects on the strength of our community, and shares why her family has decided to match all gifts made to PPMD up to $50,000 this holiday season. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 15, 2015 Category: Neurology Source Type: news
The Drisapersen Ad Comm: Where We Are Today
Read PPMD’s latest blog to learn where things stand from our perspective since the drisapersen Advisory Committee Meeting as the community waits for a decision. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 15, 2015 Category: Neurology Source Type: news
Marathon Expanded Access Program for Deflazacort: FAQs
These FAQs explain what we have learned so far regarding Marathon Pharmaceuticals and their expanded access program for deflazacort in the US. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 14, 2015 Category: Neurology Source Type: news
PPMD Houston Roundtable: Session Recordings
In October, PPMD held a FACES Roundtable in Houston that included an incredible line up of global experts in care and research. In an effort to ensure this information reached families beyond Houston, we recorded the sessions (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 11, 2015 Category: Neurology Source Type: news
PPMD's Pat Furlong Receives FDLI Distinguished Service and Leadership Award
Congratulations to PPMD President Pat Furlong on being named a recipient of the 2015 FDLI Distinguished Service and Leadership Award! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 10, 2015 Category: Neurology Source Type: news
BIO and PPMD Launch Initiative to Share Best Practices on Patient Preference Studies
PPMD and the Biotechnology Industry Organization (BIO) are proud to announce the launch of a new collaboration designed to share best practices for the development of disease-specific patient preference studies. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 9, 2015 Category: Neurology Source Type: news
Expanding the Duchenne Therapeutic Opportunities by Targeting Myostatin
Our Duchenne community is well aware of the need for combination therapies. We are all hopeful that by combining several targeted therapies, we could end Duchenne, stop progression for every single individual. Regeneration of muscle and improving muscle fiber size is thought to be an important piece of the puzzle. Learn more about the current status of research & clinical trials targeting Myostatin (one of the regulators of muscle regeneration) in PPMD's blog. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 8, 2015 Category: Neurology Source Type: news
Register for PPMD's 2016 Advocacy Conference in Washington, DC (February 21-23, 2016)
PPMD's Annual Advocacy Conference, held in the first quarter of each year, is the only officially organized gathering on Capitol Hill for the Duchenne community. Families affected by Duchenne will storm Capitol Hill on February 22nd and 23rd to meet with members of Congress and their staffers asking them to focus federal resources on Duchenne. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 4, 2015 Category: Neurology Source Type: news
Every gift doubled--for every single family.
We're all in this together. Every single success. Every single setback. Every single one of you. Donate today and your gift will be matched dollar for dollar -- helping to fund promising early-stage research that has the potential to benefit every single person with a Duchenne diagnosis. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - December 2, 2015 Category: Neurology Source Type: news
Take a moment today
Today, PPMD is thankful for you and your family, and we promise to continue to do everything possible until we end Duchenne for every single one of you. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 26, 2015 Category: Neurology Source Type: news
A Historic Day for Duchenne
PPMD President Pat Furlong reflects on today's Advisory Committee meeting for BioMarin:
"This community should be proud - proud of every person in this community who stood up at that microphone today in a room full of decision makers and told their story, spoke their truth (and shared their lived experience of this diagnosis). Proud of every person who submitted written testimony asking the agency to consider an approval. But most importantly, we should be proud of the incredibly brave young men and their families who participated in this trial. They are our strength. And they are why we continue to work tirelessly to end ...
Source: Parent Project Muscular Dystrophy - November 25, 2015 Category: Neurology Source Type: news
November 24: FDA Advisory Committee Meeting - Live Webcast Information
November 24th is a historic day for the Duchenne community as we enter into our first Advisory Committee Meeting with the FDA. Make sure to watch a live stream of the Ad Comm, or follow @ParentProjectMD on Twitter, using the hashtag #DuchenneApprovals for regular live updates. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 23, 2015 Category: Neurology Source Type: news
Webinar Recording: Understanding Myostatin Inhibition
Watch our webinar recording to learn more about the importance of myostatin inhibition research. Sponsored by educational partners Bristol-Myers Squibb and Pfizer, this webinar helped to put context and clarity around this treatment pathway so families have a better understanding as they see clinical trials in this area. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 23, 2015 Category: Neurology Source Type: news
The Big Day is Almost Here!
Here’s the latest update on next week’s Ad Comm including logistical details for those attending in person, as well as how to watch a live stream from home. If you are attending, PPMD has worked closely with the FDA to make sure people needing extra assistance are comfortable throughout the day. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 18, 2015 Category: Neurology Source Type: news
FDA Advisory Committee Scenarios
Visit our blog to read more about what we have learned about the FDA Advisory Committee process and watch our webinar recording. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - November 17, 2015 Category: Neurology Source Type: news
