The committee is meant to represent the teen and adult voice of people living with these conditions, and to help their patient community by engaging in advocacy, education, mentorship, and awareness activities. It also ensures that their voices are reflected in PPMD’s legislative, policy, and program activities.
Applications must be submitted by Nov. 30.
To be eligible, members must be 18 or older at the time of application and diagnosed with either Duchenne or Becker, two progressive muscle-wasting conditions caused by mutations in the DMD gene.
When such mutations result in no production or small amounts of a functional dystrophin protein, patients develop Duchenne, while those resulting in insufficient levels or less-efficient dystrophin lead to Becker muscular dystrophy, with milder symptoms and slower progression. Notably, dystrophin is a key protein in muscle fiber growth and protection.
PAAC members, serving in a formal advisory capacity for their respective term, are required to participate in monthly committee Zoom meetings.
Also, they typically start the year by participating in PPMD’s Advocacy Conference, in Washington, D.C. The conference provides advocate training to patients and their families before meeting with members of Congress to ensure they understand the urgency and need for federal focus and funding on Duchenne care and research.
This years’ conference focused on two main priorities: to encourage members of Congress to support Duchenne-specific funding and programs at national health agencies, and to have patients’ voices and experiences incorporated into the U.S. Food and Drug Administration’s decision-making tool, called the Benefit-Risk Framework.
PAAC members then usually help to plan the Teens & Adults Track for PPMD’s Annual Conference each June — the largest, most comprehensive, international conference focused solely on Duchenne. Due to the COVID-19 pandemic, this year’s annual conference was held virtually.
Other in-person meetings are occasionally held; meeting attendance by PAAC members is strongly encouraged, but not required for membership. Members are estimated to commit roughly five hours per month to PAAC activities.
To apply for the 2021 PAAC, patients must complete and submit a membership application, which includes a resume request that can be submitted through the application or sent to Pat Moeschen ([email protected]). PPMD emphasizes it will consider all applicants, even if lacking experience.
Current members will hold a Zoom meeting on Wednesday (Nov. 4) at 7 p.m. EST to provide more information about PAAC membership and answer questions from potential applicants. Click here or send an email to Pat Moeschen by Wednesday to register.
Participation in the informational meeting is not required for applicants to be eligible.
The PAAC also launched a Facebook group where Duchenne and Becker patients (and their families) can discuss important daily-life topics, and where the committee shares its perspectives and routinely posts educational materials.
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