The heart is a muscle too. And PPMD has been asking questions about the heart for many years:

• What do we know and what do we need to learn about heart disease (cardiomyopathy)?
• If treatments protect skeletal muscle, is the heart ‘built’ to last?
• How early should cardio-protective medicines be started?
• What about new heart failure medicines such as SGLT2 or Entresto?
• Are individuals with Duchenne, Becker, and female carriers likely to have arrhythmias?
• How do we make sure our HEARTS stay strong with every beat?

PPMD is committed to our cardiac initiative and these are only a few of the questions we are asking pediatric and adult cardiologists. We have to learn everything we can about the heart, how to preserve and protect heart function, and how best to treat the heart to improve the outcomes of people living with cardiomyopathy (heart failure). We have to evaluate how new Duchenne therapies impact the heart, how we can measure cardiac disease progression, and when to consider using new heart failure medicines and devices such as LVAD.

PPMD’s Ongoing Cardiac Initiative

PPMD has invested nearly $7 million in this effort, most recently awarding a $2 million grant to the Advanced Cardiac Therapies Improving Outcomes Network (ACTION). This network facilitates the collaboration of cardiologists from across the country to focus on optimizing care and filling gaps in knowledge. 

On the heels of this recent grant last year, PPMD hosted the 2022 Cardiac Care and Duchenne – Professionals Workshop with over 80 cardiologists, neuromuscular specialists, researchers, and industry partners. Led by Dr. Chet Villa (Cincinnati Children’s Hospital), Dr. Deip Nandi (Nationwide Children’s Hospital), and Dr. Linda Cripe (Nationwide Children’s Hospital), the group spent two days discussing how to harmonize current guidelines in caring for the heart and advocate for closer surveillance of the heart in clinical trials of investigational therapies such as gene therapy, and adapting treatments following the approval of new therapies.

Download the Report

Our goal is to promote transparency and share learnings with the community as we gather new information. PPMD’s 2022 Cardiac Care and Duchenne – Professionals Workshop Report describes the results of the meeting, designed to improve care and treatment of the heart.

Download the Report >

Upcoming 2023 Professionals Workshop

This week, PPMD will host a follow up 2023 Cardiac Care & Duchenne – Professionals Workshop with an expanded group of clinicians and researchers to advance earlier discussions into areas such as:

• New cardiac medications to be used in Duchenne and Becker
• How to care for female carriers
• The use of wearable and remote technology to measure heart function
• Post gene therapy surveillance

We will share an update following this meeting and will continue to keep you informed as we work to ensure the hearts of those we love stay stronger with every beat.