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.
MDF and its international team of clinical care partners just met in Miami to draft consensus-based Care Considerations for doctors, pharmaceutical companies and federal regulators reviewing potential therapies. Read more in this article.
MDF's Interim Chief Science Officer, Dr. John Porter, brings a wealth of experience to MDF as a former academic researcher, program director at the National Institutes of health (NIH) and Chief Executive Officer of a patient advocacy organization. John offered his thoughts recently on the importance of patient advocacy in drug development.
Researchers from the University of Costa Rica and the University of Glasgow teamed up to investigate the DNA mutation causing myotonic dystrophy type 1 (DM1). They found that polymorphism in the MSH3 mismatch repair gene is associated with the levels of somatic instability of the expanded CTG repeat in the blood DNA of myotonic dystrophy type 1 patients.