Following decades of strategic advocacy, research investment, and biopharmaceutical development, the U.S. Duchenne community recently arrived in the post-approval space and is poised to receive additional novel therapies from a robust innovation pipeline.

Parent Project Muscular Dystrophy (PPMD) is deeply engaged on behalf of families nationwide as industry sponsors work with the Food & Drug Administration (FDA) to navigate the critical regulatory steps. In addition, PPMD is working now within the access environment to help ensure that patients and caregivers have timely access to these disease-modifying medications.

As part of our effort, PPMD is hosting the Access to Emerging Therapies Forum on February 13th, 2017 for our patient community and relevant stakeholders. Being held in connection with PPMD’s Annual Advocacy Conference, our presenters include policy experts who will present on the various points on the access continuum including:

• Describing how individuals might obtain access to candidate therapies under study (experimental therapies)
• Familiarizing community members with the fundamentals of how approved therapies enter the marketplace and are made available through health insurers (approved therapies)
• Discussing key policy issues impacting access to innovative therapies (policy issues)

It is our hope that the Forum will be an important step in helping to continue to empower the Duchenne community and our partners to navigate the regulatory and approval pathways necessary to achieve desired levels of clinical treatments and care appropriate for themselves/their children.

Learn more

PPMD has assembled resources that will help families and medical providers at each stage of the healthcare access process. Click here to view a road map aimed at guiding you through this complex process – complete with sample letters and links to relevant publications and other resources.