MDF held the first myotonic dystrophy Patient-Focused Drug Development meeting with key senior leadership from the Food and Drug Administration (FDA) as part of the 2016 MDF Annual Conference. We designed the meeting to build on the work MDF has been conducting with the FDA to define and optimize the regulatory pathway for potential DM therapies. The meeting, the first Externally-Led PFDD meeting and the largest PFDD meeting held to date with over 200 attendees in the room and online, is helping change the way the FDA understands DM, the DM community and the approach to regulatory review for DM therapies.
This year’s closed Professionals Track includes sessions and meetings specifically for academic and industry professionals, including a Drug Development Roundtable, updates on MDF-funded assay and screening, endpoints and new animal model development programs, and a DM Clinical Research Network.
The Jensen family hosts an incredible crawfish boil each summer to support Care and a Cure. This year’s boil was bigger than ever with San Diego Harley-Davidson as a partner. To date, this event has raised nearly $100,000! Read more here.
MDF recently participated in a European Union workshop to plan a network for research and care in DM1. We constructively contributed to the workshop, while emphasizing the international activities that MDF already has underway and the need for coordinated efforts as the only path forward to improve the quality of life and availability of treatments for those living with DM.
Our Staff recently attended the annual BIO convention, the meeting and partnering place for the biotechnology and pharmaceutical industry. BIO is about opportunities—opportunities for learning and working together to foster new drug development. MDF engaged key industry leaders in discussions of therapy development in DM, exchanging information on existing DM drug discovery and development, as well as opportunities for launching new DM programs.