What is an Ad Comm?
Ad Comm. It’s a term our community will be hearing a lot this fall as companies get closer to the possible approval of treatments for Duchenne. But what is an Ad Comm and how does it involve and affect you or your child? PPMD will host a webinar on September 2 at 1pm eastern to discuss this topic in detail. Guests will include FDA staff. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 12, 2015 Category: Neurology Source Type: news
Imperatives for DUCHENNE MD: A simplified guide to care for Duchenne published in PLOS
PPMD is proud to present a published version of Imperatives for DUCHENNE MD a one page snapshot of Duchenne care created by a small group of international experts from PPMD, UPPMD, and TREAT-NMD. We hope you continue to find this a useful resource and we thank PLOS for helping us share this information with the medical community. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 12, 2015 Category: Neurology Source Type: news
One month away from World Duchenne Awareness Day!
People around the world will help participate in the second annual World Duchenne Awareness Day on September 7th. There are a variety of ways you can get involved with this international effort how will you raise awareness in your community? (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 8, 2015 Category: Neurology Source Type: news
PPMD Submits Comments on FDA's Draft Guidance on Duchenne
We are coming to the end of the 60 day open comment period for the FDA’s draft guidance on Duchenne. PPMD submitted our comments to the agency yesterday. While we hope you agree with our response, we respect alternate viewpoints. If you disagree with any comments or have other issues you think the guidance needs to address, we urge you to submit your opinions as well through the FDA public comment mechanisms. This community-wide effort continues to be acknowledged as precedent setting and a variety of rare disease groups are following in our footsteps. We have so much to be proud of and look forward to next steps with th...
Source: Parent Project Muscular Dystrophy - August 7, 2015 Category: Neurology Source Type: news
Action ALERT: Urge your Senators to Cosponsor S. 1597, the Patient-Focused Impact Assessment Act
Call and email your Senators today and urge them to sign on as a cosponsor of S. 1597, the Patient-Focused Impact Assessment Act, before they go on recess on August 8th. This is critical legislation for our community. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 6, 2015 Category: Neurology Source Type: news
Different Together: PPMD Announces the Duchenne Regulatory Science Consortium
PPMD is thrilled to announce the launch of the Duchenne Regulatory Sciences Consortium (D-RSC) with the Critical Path Institute! Each instance of Duchenne seems to be as unique as the person it affects. The D-RSC will begin by aggregating a wide array of clinical data for analysis by consortium members, with the goal of developing a disease progression model that can be used in the development of new treatments, and reduce the amount of time it takes to get them to people living with Duchenne. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 4, 2015 Category: Neurology Source Type: news
Changes to Deflazacort Ordering Process (via Masters Pharmaceuticals)
Masters now has an increasing supply of Deflazacort (a steroid available to treat Duchenne outside the US). However, due to recent regulatory changes, changes have been made to the ordering process to assure that the company is able to continue to supply Deflazacort to US families. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - July 28, 2015 Category: Neurology Source Type: news
PPMD's 2015 Connect Conference Resources & Recordings
Presentations from PPMD ’s 21st Annual Connect Conference in Washington, D.C. are now available for download. < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=ZkZbikei9as:JmaFcIrZlG0:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=ZkZbikei9as:JmaFcIrZlG0:qj6IDK7rITs" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=qj6IDK7rITs" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=ZkZbikei9as:JmaFcIrZlG0:V_sGLiPBpWU" > < img src="http://feeds.feedb...
Source: Parent Project Muscular Dystrophy - July 22, 2015 Category: Neurology Source Type: news
PPMD's 2015 Connect Conference Resources & Recordings
Presentations from PPMD’s 21st Annual Connect Conference in Washington, D.C. are now available for download. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - July 22, 2015 Category: Neurology Source Type: news
BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Amenable to Exon 51 Skipping
The Duchenne community should celebrate. Today, Biomarin submitted the rolling NDA on drisapersen. This means we now have a second NDA submitted for Duchenne. Santhera is likely to follow soon. By first quarter 2016, we may see 3 approved drugs with more to follow. This is exactly what we have been working for, hoping for. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 30, 2015 Category: Neurology Source Type: news
PPMD Awards $148,000 Grant to University of Washington
Exon skipping and gene replacement are leading candidate therapeutics in the Duchenne space right now. It is our hope that Dr. Froehner’s project will help improve decision making in the design and targeting of both exon skipping oligonucleotides and gene therapy vectors so that patients receive the best therapy possible and the chances of approvals in a timely manner are increased. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 30, 2015 Category: Neurology Source Type: news
Webinar Recording: FOR-DMD Study
The recording of our webinar on the FOR-DMD study (developing standardized corticosteroid treatment for Duchenne) is now available. Included in the webinar is an overview of the aims of the FOR-DMD study, along with a discussion of why the study is particularly relevant in regard to current/future Duchenne clinical trials. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 30, 2015 Category: Neurology Source Type: news
End Duchenne eNews: PPMD Launches Robotics Initiative
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 - April 30, 2015 Category: Neurology Source Type: news
Webinar Recording & Summary: Orthopedic Complications in Duchenne
If you missed our webinar on Orthopedic Complications in Duchenne (covering contractures, scoliosis, & fractures), you can now access a recording of the webinar online. Summary notes and responses to additional questions we received during the webinar are also available. < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=vuYtfMaxDGg:B2nyXlzQIL0:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=vuYtfMaxDGg:B2nyXlzQIL0:qj6IDK7rITs" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=qj6...
Source: Parent Project Muscular Dystrophy - April 30, 2015 Category: Neurology Source Type: news
Webinar Recording & Summary: Orthopedic Complications in Duchenne
If you missed our webinar on Orthopedic Complications in Duchenne (covering contractures, scoliosis, & fractures), you can now access a recording of the webinar online. Summary notes and responses to additional questions we received during the webinar are also available. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - April 30, 2015 Category: Neurology Source Type: news
