Publication of a new systematic review provides insights into clinical outcome measures for DM2.
Induced pluripotential stem cells (iPSCs) from DM2 provide new tools for CNS-focused mechanistic and translational studies.
A new publication finds a complex mechanism of action of furamidine in DM1 models.
As a young cardiologist, Dr. Denis Duboc didn’t think it made much sense that his fellow specialists would treat patients with myotonic dystrophy (DM) and other muscle diseases as if they were treating other cardiology patients. Since becoming a researcher, his work has focused on better ways to provide care for DM patients.
David Berman was abruptly introduced to myotonic dystrophy when his wife Sarah gave birth to their daughter Zoé in December 2007. Doctors were immediately concerned with her floppiness and inability to feed. For the first few days of her life, Zoé was fed with formula in a tiny tube that ran along one of her parents’ fingers and into her mouth. This still required enormous effort by Zoé, and the doctors feared that feeding might be burning more calories than it provided.
Peer-reviewed Medical Research Program (PRMRP) consumer advocate Suzette Ison recently participated in the evaluation of research applications submitted to the PRMRP sponsored by the Department of Defense. Suzette was nominated for participation in the program by the Myotonic Dystrophy Foundation, located in San Francisco, Califronia. As a consumer reviewer, she was a full voting member, (along with prominent scientists) at meetings to help determine how the $330 Million appropriated by Congress for Fiscal Year 2018 will be spent on PRMRP research.
Close monitoring of respiratory function reduces respiratory complications in DM1.
MDF has released a Request for Applications for projects that address the evaluation of genome editing strategies for DM1. The focus of this RFA is early stage discovery and development of in vivo genome editing technologies. Up to two awards of $250,000 each are planned.