Registration for PPMD Advocacy Conference Now Open

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by Vanessa Pataia |

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Duchenne advocacy

Registration for the upcoming Parent Project Muscular Dystrophy (PPMD) Advocacy Conference is now open.

During the conference, which will be held March 8–10 in Washington, D.C., participants will receive extensive training and support to empower and prepare them to advocate for Duchenne muscular dystrophy directly with members of Congress.

Information about registration, contacts and accommodations is available here.

PPMD advocates visit Washington every year to ensure the agencies with the power to influence Duchenne research and care programs — such as the Centers for Disease Control and Prevention (CDC), the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA) and the Department of Defense (DoD) — are appropriately funded and have the specific report language to guide them in making decisions about Duchenne programs and priorities.

Owing to the their presence in Washington, advocates are able to make sure  lawmakers better understand the clinical, research, and regulatory priorities of those living with Duchenne.

By advocating for Duchenne in this way for the last two decades, more than $600 million in funding has been awarded specifically to Duchenne research and care programs at the NIH, CDC, and DoD, according to PPMD.

It also has supported the development of Duchenne therapies, with more than 30 companies having potential Duchenne treatments in their pipelines.

In addition, the establishment and updating of standard-of-care for everyone diagnosed with Duchenne has improved the quality of life and extended lifespan significantly, according to the non-profit organization.

This year’s conference will start with advocate training for all participants. A special event discussing federal agency involvement in Duchenne will follow on March 9.

Over the rest of the conference, advocates will meet with members of the Senate and House of Representatives to advocate for two main priorities.

The first priority will be to encourage members of Congress to support Duchenne-specific funding and programs at the CDC, NIH, FDA and DoD with the potential to affect Duchenne.

The second priority will be to have patients’ voices and experiences incorporated into the FDA’s decision-making tool, called the Benefit-Risk Framework.

Advocates will focus on requesting passage of the BENEFIT Act (Better Empowerment Now to Enhance Framework and Improve Treatments Act). That would allow for patient experience data — such as patient reported outcomes, patient preferences, and data from interviews, testimonials, and focus groups — to be included in the FDA’s benefit-risk evaluation of potential therapy. 

Ultimately, it would mean that the patient’s perspective would influence the FDA’s decisions.

According to PPMD, the event is an opportunity to continue changing the landscape in the fight to end Duchenne.