A Historic Day for Duchenne
PPMD President Pat Furlong reflects on today's Advisory Committee meeting for BioMarin.
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.
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.
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.
End Duchenne eNews: Tackling Duchenne from Every Angle
Catch up on the latest research and community updates in this month's End Duchenne eNews.
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.
PPMD Supports Advancing Targeted Therapies for Rare Diseases Act of 2015 (S. 2030)
For over a year, PPMD has been working with congressional champions and industry partners to help lead an effort to streamlining the regulatory pathways and review processes for targeted therapeutics (which would include such things as 'follow on exons') by clarifying the FDA’s existing authority to leverage data previously used in the approval of a targeted product when approving a new therapy.
Webinar Recording: Decode Duchenne Genetic Testing Program
Watch our webinar recording to learn how the Decode Duchenne program works, who can apply, and why genetic testing is so important for you and your loved ones.
Help Us Protect Your Wheelchair Technology!
The Centers for Medicare and Medicaid Services (CMS) has announced that it will apply Medicare competitive bid program pricing to Complex Rehab wheelchair accessories effective January 1, 2016; an action that will likely reduce access to wheelchair features many of our families depend on.
A Paradigm Shift in Drug Discovery
The Critical Path Hill Briefing that was just held demonstrated the importance of moving from “if and when [scientists and companies] are willing to share data” to an “institutionalized sharing of data.”
PPMD Submits Results of Patient-Centered Benefit-Risk Assessment Study in Duchenne & Becker to FDA
PPMD is pleased to announce that late yesterday we submitted the results of our most recent patient-centered benefit-risk assessment study to the FDA. Based on this study, we are hopeful the FDA will have a better understanding of preferences of Duchenne patients and caregivers in regards to a specific pulmonary therapeutic target.
How You Can Participate in the Upcoming FDA Advisory Committee Meetings
With the recent announcement of Advisory Committee (Ad Comm) dates for both BioMarin and Sarepta, families in our community are eager to be engaged and understandably have many questions. Here is a quick guide to what information has been confirmed and how you can participate.
Ad Comms Update: Group Letter Submitted to FDA
PPMD is proud to help author and share the attached letter to Dr. Janet Woodcock at the FDA. Along with CDMD at UCLA, the Duchenne Alliance, and The Race to Yes, PPMD is asking Dr. Woodcock to consider moving Sarepta’s Advisory Committee date to the same week in November as BioMarin’s.
Ad Comms Update: An Overwhelming Day
We all have many questions about what the January Ad Comm date for Sarepta, after the PDUFA date for BioMarin may mean. And PPMD is working with our regulatory experts to seek those answers and to understand how we can continue to work together as a community to ensure that both products are offered regulatory pathways to success so that patients ultimately may have access to either product, if approved.
PPMD’s PRO Project: Adding more tools to help with upcoming clinical trials in Duchenne
Patient-report outcomes (PROs) are an important tool that allow patients and families to provide structured input about their own experiences and can be used in clinical trials and other research to help measure meaningful differences over time. Our community needs more meaningful PROs so that we have as many tools in our toolbox to help with upcoming clinical trials. That’s why PPMD wants to take a deeper dive into what is most important to you -- what would you value and expect as a result of a new therapeutic?
PPMD Urges Senate to Move Forward on #Cures2015 Innovation for Healthier Americans Bill
PPMD continues to urge Members of Congress to support the Patient-Focused Impact Assessment Act (PFIA) as part of the Innovation for Healthier Americans bill. Please help us ensure that all Senators are aware of PFIA by reaching out today.