by: Parent Project Muscular Dystrophy
The approved therapy landscape has changed drastically in Duchenne, especially over the last year. With multiple newly approved therapies comes a lot of questions about how to navigate these drugs: when they are prescribed; who are the right patients for each drug; and how to navigate this ever-evolving and increasingly complex landscape.
During the “Navigating the Approved Therapy Landscape” session at PPMD’s 30th Annual Conference in June 2024, panelists first gave an overview of three recently approved therapies, AGAMREE, DUVYZAT, and ELEVIDYS. These overviews aimed to provide the community with a better understanding of what the drugs are, how they work, and who the patient population is for each of them.
The second portion of the session featured expert clinicians Aravindhan Veerapandiayn, MD, from Arkansas Children’s Hospital; Paula Clemens, MD, from the University of Pittsburgh; Omer Abdul Hamid, MD, from Nemours Children’s Hospital; Crystal Proud, MD, from Children’s Hospital of the King’s Daughters; and Diana Bharucha-Goebel, MD, PhD, from Children’s National Hospital. The group dove into a lively discussion around decision making, incorporating the patient and caregiver voice, and the complexities of prescribing multiple approved therapies for an individual.
Approved Therapies Discussion Continues in Part 2
The conversation around approved therapies continued in the “Current Approved Therapies Panel Discussion Pt. 2: Broad Access to Gene Therapy” session. This follow-up discussion with clinicians Emma Ciafaloni, MD, from University of Rochester; Kevin Flanigan, MD, from Nationwide Children’s Hospital; Aravindhan Veerapandiyan, MD, from Arkansas Children’s Hospital; Richard Shell, MD, from Nationwide Children’s Hospital; Larry Markham, MD, from Children’s Health; and Carol Wittlieb Weber, MD, from Children’s Hospital of Philadelphia, focused on shared decision-making regarding the newly expanded ELEVIDYS label, and what this expanded label means given that it is broadly applicable for the majority of individuals living with Duchenne.
While these newly approved therapies offer exciting opportunities to many patients, it’s important to discuss with your neuromuscular team which treatment options you/your child may qualify for and help guide you through the decision making process.
PPMD For You
If you still have questions, PPMD is here for you. Through our PPMD For You program, you can schedule a one-to-one meeting with an expert member of the PPMD team for personalized support.
Access & Coverage Resources
Additionally, we know access and coverage for approved therapies, appointments, procedures, & equipment can sometimes be challenging. To make this process easier, PPMD has assembled resources to help families and medical providers at each stage of the access process. Our roadmap aims to guide you through every stage and features sample letters, links to publications and other resources, a list of things to consider, and tips for advocating for access.
You can also schedule a one-to-one Navigating Resources and Benefits appointment through the PPMD For You program, which guides those who are looking for support resources and/or want to discuss navigating benefits or access programs.
