Action Alert! Urge Congress to Support Progress in the Fight to End Duchenne

Urge your House and Senate Members to Support the FY 2024 Duchenne Funding Request & BENEFIT Act

It’s that time of year again. Members of the House and Senate are working, right now, to develop and submit their policy priorities for the annual spending bills that will fund the NIH, CDC, DOD, FDA, and other key federal agencies for 2024.

We need you to contact your House and Senate members as soon as possible and urge them to continue making Duchenne patient care, public health, and research a top priority. Click here to learn more about this year’s request to Congress.

Please fill out the information below, there is an optional space for you to fill in your personal story within the body of the message.

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Support the FY 2024 Duchenne Appropriations Request and the BENEFIT Act

Dear [Decision Maker],

In 2001 Congress enacted the Muscular Dystrophy Community Assistance, Research and Education or MD CARE Act, which dramatically transformed efforts to combat Duchenne and other forms of Muscular Dystrophy. As a result of this Act and subsequent amendments, federal commitments to research expanded, helping spur scientific breakthroughs to develop potential therapies. These commitments have also leveraged significant non-federal funding from academic institutions, industry, and venture investors in a true public-private partnership model. In addition to research breakthroughs, the MD CARE Act has helped standardize patient care. These care standards have helped markedly lengthen and improve the lifespan of the average Duchenne patient.

Achieving future success requires the continued support of programs and policies to support Duchenne research, public health and drug development efforts. We need your help. To support this work, our first request is for you to please sign onto the annual Duchenne appropriations sign-on letter being led by Senators Roger Wicker (R-MS) and Debbie Stabenow (D-MI) in the Senate and Congresswoman Doris Matsui (D-CA) and Congressman Troy Balderson (R-OH) in the House.

This letter seeks support for key Duchenne programs and policies including:

$8 million for CDC's Muscular Dystrophy Program funding and requests CDC evaluate the impact of Care Considerations as identified in the recent report to the Committee.
$15 million for the Duchenne Muscular Dystrophy Research Program within DOD's Congressionally Directed Medical Research Programs (CDMRP)
Requests NIH produce a progress report on the Wellstone Centers
Requests FDA support the development of patient experience data to inform clinical research design and regulatory reviews.
Requests HRSA recommend expedited consideration of Duchenne for newborn screening and incorporation of the patient community voice in the review.

I urge you to sign onto the FY24 letter which means so much to me and my family. To sign on to the Senate letter, please contact Sally Farrington in Senator Wicker's office or Julia Johnston in Senator Stabenow's office.

To sign the House letter, please contact Jackie Weinrich ( with Rep. Matsui or Megan Porter ( with Rep. Balderson.

Our second request to Congress is to support the BENEFIT Act. While much progress has been made in Patient-Focused Drug Development, some significant gaps remain. One such gap is the lack of any requirement in law today that the FDA includes as part of its risk-benefit framework any patient experience or patient-focused drug development (PFDD) data. This means that the agency's signature tool for evaluating risk-benefit does not have to data from the patient perspective that could be critical to informing the agency's evaluation and, ultimately, the decision on whether or not to approve a product. To address this gap, Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN) have introduced S. 526 the Better Empowerment Now to Enhance Framework and Improve Treatments or the BENEFIT Act. A companion bill has been introduced by Representatives Matsui and Wenstrup in the House (H.R.1092). This legislation will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, PFDD and related data including information developed by a product sponsor or a third party such as a patient advocacy organization or academic institution be considered as part of the risk-benefit assessment. This action will send an important signal to all stakeholders that patient experience and PFDD data will be fully incorporated into the agency's review process and will encourage such entities to develop scientifically rigorous and meaningful tools and data. The BENEFIT Act will continue this evolution by filling a sizeable gap by ensuring such data is fully considered as part of the FDA's risk-benefit assessment for any new products. Advance patient engagement by cosponsoring the BENEFIT Act today.

In the Senate, you can support S. 526 the BENEFIT Act by contacting either Senator Wicker or Senator Klobuchar's office. In the House, you can support H.R. 1092 by contacting either Representative Matsui or Representative Wenstrup.

We appreciate your consideration of these two requests.

Thank you for all you do.

[Your Name]
[Your Address]
[City, State ZIP]
[Your Email]