For those of us who’ve been in this community for decades, at some point we began to believe that we’d never see the day when products that had been designed specifically with the intent to treat individuals with Duchenne would be reviewed by the FDA. But here we are—standing on the shoulders of the community members who helped blaze this trail—and knowing that this coming week signifies the first of MANY products coming through the robust and promising pipeline that our community and our partners continue to move forward.
So, whether you are attending the Advisory Committee Meeting (Ad Comm) for BioMarin in-person in White Oak next week, will be watching via the live stream, or will plan to attend a future product review, our PPMD team has worked closely with the team at the FDA over the last several months to ensure that our community would have access to this proceedings and will be able to participate fully. Here are some important logistical updates and details:
For Those Attending In-Person
PPMD is hosting a training session for all those participating in the Open Public Hearing. The training will be from 4-6 on Monday, 11/23.
For more details and to RSVP, please contact Ryan Fischer at firstname.lastname@example.org
PPMD, the Every Life Foundation, and CureDuchenne are hosting a dinner on Monday, 11/23 from 6-8 pm for community members who are in town for the Ad Comm. For more information or to RSVP, contact Julia Jenkins at the EveryLife Foundation at email@example.com
The Ad Comm meeting will be held on November 24, 2015, from 8 a.m. to 5:30 p.m, at the FDA White Oak Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room (Rm. 1503), Silver Spring, MD 20993-0002.
On the day of the event, all meeting attendees will be required to enter through Building 1 on FDA’s Main Campus and go through the security screening. PPMD has worked with the FDA to ensure that security will be equipped to effectively and efficiently screen all community members, and those utilizing mobility equipment will have alternate screening options and will not be required to go through the x-ray machine.
More information about attending meetings on the FDA campus can be found at the FDA website.
It is requested that all individuals with disabilities be dropped off at the entrance to Building 1, rather than using the FDA campus shuttle service.
While it is not required that members of the Duchenne community remain at the FDA for the full duration of the Ad Comm’s proceedings, we recognize that many may opt to stay for the entire day. Therefore, we have worked to make arrangements to add to your comfort throughout the day:
For Those Participating Remotely
Live Stream Access
The link to the live stream will be made available on either Friday or Monday and will be able to be accessed on the
UPDATE: Live stream information has now been made public. Click here to view.
For more information about the FDA Ad Comms, please visit the links and resources that we at PPMD have compiled over the past few months with the assistance of our community members, regulatory advisors, and federal agency partners: