Every year our community heads to Washington, D.C. as part of PPMD’s Advocacy Conference (this year, being held virtually on March 10th — and there’s still time to register!) to communicate to Congress the need for continued funding for the federal agencies that impact Duchenne. Along with the funding, we also request that Congress include Duchenne-specific “report language” which includes specific priorities and guidance for Duchenne-related spending.

With PPMD’s advocacy season in full swing, we hosted a series of webinars during discussing two federally funded research studies in Duchenne, highlighting the importance of federal investment the muscular dystrophies and of advocacy engagement by our community.

Weekend Steroids and Exercise for Duchenne

Wednesday, March 3 at 1 PM EST

Dr. Taivassalo joined us discuss the University of Florida’s new Department of Defense funded study to evaluate the potential of delaying disease progression and strengthening muscle function in patients with Duchenne by combining a low dose weekend steroid regimen with exercise training. This study is supported by PPMD and the Department of Defense Duchenne Muscular Dystrophy Research Program (DMDRP).

Moderator: Eric Camino, PhD
Vice President, Research and Clinical Innovation
Parent Project Muscular Dystrophy

Presenter: Tanja Taivassalo, PhD
Associate Professor
Department of Physiology and Functional Genomics
University of Florida

Assessing the Risk of Arrhythmias and Sudden Cardiac Events in Duchenne

Thursday, March 4 at 1 PM EST

Cardiac involvement in Duchenne starts in early childhood, and by the end of the second decade, weakening of the heart muscle is observed in >40% of men and can lead to heart failure, heart rhythm abnormalities and sudden cardiac death. Andreas Barth, MD, PhD and Adityo Prakosa, PhD joined us to discuss a multicenter trial to better assess the risk of arrhythmias in patients with Duchenne.

The role of ambulatory EKG monitors (Holter and Event monitors; implantable loop recorders), cardiac imaging (cardiac MRI) and novel computational technologies (computer simulations using patient-specific 3D heart models) for risk stratification of sudden death were discussed. This study is supported by PPMD and the Department of Defense Duchenne Muscular Dystrophy Research Program (DMDRP).

Moderator: Rachel Schrader, MS, APRN, CPNP-PC
Vice President, Clinical Care & Education
Parent Project Muscular Dystrophy

Presenter: Andreas Barth, MD, PhD
Director, Center for Inherited Heart Diseases
Johns Hopkins University School of Medicine

Presenter: Adityo Prakosa, PhD
Associate Research Scientist
Alliance for Cardiovascular Diagnostic and Treatment Innovation (ADVANCE)
Johns Hopkins University