Research

MDF has awarded new grants to develop phenotypic mouse models of DM as well as new DM1 and DM2 cell lines. Read more about these new partnerships.

A new study from Dr. Dimitri Renard and his team applies metabolic imaging to identify brain regions affected in DM1. Read about their findings here.
 

MDF and the MDF UK 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.

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.

“Participating in a research study will help the greater good, and I’m hoping one may help me too,” says Caroline Easterling about why she participates in DM research. Read on for Caroline’s advice for participants.

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.

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.

A former MDF Fellow, Dr. Yao Yao, has made a breakthrough in understanding how muscle stem cells are directed to aid muscle regeneration. Read about his important research here.