As part of our ongoing Cardiac Initiative, PPMD is proud to host the upcoming professional meeting, “Expanding Access to Heart Transplantation: Focus on Muscular Dystrophy,” taking place September 27-28, 2024, at Stanford University. This important event will bring together leading experts in cardiology, neuromuscular disorders, and transplant medicine to address a critical but often overlooked issue in the Duchenne community—access to heart transplantation.

Changing Hearts and Minds

As we all know, Duchenne is a genetic condition that affects the muscles throughout the body, including the heart. In fact, heart problems are often a leading factor in overall health decline for individuals with Duchenne. Despite this, cardiac transplantation – a potentially life-saving procedure – is rarely considered an option for Duchenne patients.

Why the hesitation? There are several factors at play:

1. Timing: Determining the optimal moment to refer a patient for transplant evaluation is complex.
2. Experience: Few medical centers have experience with both Duchenne and heart transplantation in this population.
3. Misconceptions: Some believe that Duchenne patients are not suitable candidates due to their overall condition.

We believe it’s time to challenge these barriers. A growing body of evidence suggests that carefully selected patients can benefit significantly from heart transplantation, potentially extending and improving their quality of life.

A Collaborative Path Forward

During this two-day meeting, discussions will focus on overcoming these barriers and developing actionable steps to expand access to heart transplantation for the Duchenne community. By bringing together experts from across disciplines, we aim to:

1. Establish clear guidelines for when to consider transplant referral in Duchenne patients.
2. Identify and support Centers of Excellence willing to evaluate and perform transplants for this population.
3. Develop standardized protocols for post-transplant rehabilitation tailored to Duchenne patients’ unique needs.

It’s time to change both minds and policies to ensure that Duchenne patients have access to all potentially beneficial treatment options, including cardiac transplantation. Through PPMD’s ongoing Cardiac Initiative and professional meetings, we aim to bring much-needed attention to this vital issue and advocate for changes that will improve the lives of individuals with Duchenne facing advanced heart failure. 

Stay tuned for updates from this important meeting as we continue to work together to expand access to treatment options and improve outcomes.