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06/09/2016 - 10:16am

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.

06/30/2016 - 10:27am

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.

06/30/2016 - 10:24am

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.

06/30/2016 - 10:13am

Researchers in Europe have reported findings from a large cohort, where significant numbers of patients with DM-like symptoms do not have the known DMPK or CNBP repeat expansions. Considerable progress has been made in understanding the genetics and pathogenic mechanisms of DM. However, unknown factors (other mutations, modifier genes) very likely will be critical to models of disease onset and progression, as well as for development of impactful therapies. This latest report, valuable because of the large, carefully assessed cohort, suggests that MBNL1 variants, theoretically a potential cause of DM, exist but are rare and thus unlikely a major contributor to DM.

06/30/2016 - 10:06am

MDF and Wyck jointly announce the funding of two new research projects. The projects address critical gaps in research infrastructure and clinical trial readiness and will increase understanding of the progression of DM, and provide measures to evaluate disease progression and the efficacy of candidate therapeutics.

 

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