For more than three decades, Parent Project Muscular Dystrophy (PPMD) has been committed to bringing therapies closer to, and improving care for, every person living with Duchenne and Becker muscular dystrophy. The Duchenne and Becker landscape is evolving rapidly, and guided by our promise to Honor Every Journey, PPMD is ensuring that every voice and everyone at every stage of the disease is represented in our progress. Your support this holiday season will directly power the next phase of bold, data-driven progress for all. 

Innovating Drug Development

We’re driving systemic change for those whose variants are not currently included in the drug development pipeline. 

Less than half of Duchenne patients live with genetic variants that are currently being targeted by approved therapies or therapies in development, such as exon-skipping therapies. That’s why PPMD is launching this multi-pronged effort to secure platform designation for exon-skipping therapies at the FDA. This designation would significantly reduce the time and cost required to bring therapies to individuals with less common variants. Your support today can help us move faster.

Your contribution will help PPMD convene a major stakeholder meeting, assemble data-driven evidence, and strategically advocate with regulators to demonstrate what this approach can unlock. We have the tools in front of us now, we must use them.

Image: “Based on patient data from the UMD-DMD database” Leckie, J., Zia, A., & Yokota, T. (2024). An Updated Analysis of Exon-Skipping Applicability for Duchenne Muscular Dystrophy Using the UMD-DMD Database. Genes, 15(11), 1489. https://doi.org/10.3390/genes15111489

Elevating Every Voice

Adults with Duchenne and Becker, as well as female carriers, deserve to be heard and play a central role in stakeholder and regulatory discussions. PPMD is expanding its advocacy efforts to ensure that federal agencies recognize, hear, and act on all voices of our community so that everyone has access to optimal care. PPMD is committed to continuing the important work of capturing sentiments of adults living with Duchenne about the current therapeutic landscape and expanding that work to include female carriers, an often-overlooked group that deserves an equal voice in care, and more therapeutic options.

We will continue to engage adults and carriers in stakeholder and regulatory discussion, and ensure that adult and carrier experiences are integrated into access and approval decisions.

Optimizing Outcomes

PPMD continues to fund groundbreaking work to redefine how disease progression is measured. One of the most promising tools to accomplish this is MRI fat fraction, a type of imaging biomarker that shows how muscle changes over time. This approach offers a steady, unbiased way to track disease progression. If validated and accepted by the FDA, MRI fat fraction could:

• Enable smarter, faster, and more equitable trial designs.
• Broaden clinical trial participation to include individuals who can’t complete traditional physical tests.

PPMD sees potential in supporting Dr. Sarah Kim at the University of Florida, whose project will use more than 13 years of imaging data to validate MRI fat fraction as both a biomarker and strengthen its potential use as an unbiased surrogate endpoint. 

This work would fall under PPMD’s Biomarkers Initiative, which was developed to support the discovery and validation of biomarkers that can be used to:

• Improve drug development by increasing the number of safety markers used to monitor response to investigational products and accelerate time to approval by creating surrogate markers for measuring stabilization or improvement related to investigational products.
• Enhance care of individuals with Duchenne and Becker by uncovering biomarkers that can be used to monitor disease progression or predict disease trajectory and early warning signs of complications.

 

PPMD is built on the strength of this community, and we will not rest until every member of this community has access to the therapies and quality care they need. Together, we can bring therapies, care, and hope to all, as we Honor Every Journey of every person with Duchenne and Becker muscular dystrophy.

Help PPMD Honor Every Journey: Donate Now