Located around the United States, MDA’s new network provides a foundation for the conduct of clinical trials in MMD1 and MMD2
MDA has launched a new, five-center clinical research network focused on type 1 and type 2 myotonic muscular dystrophy (MMD1 and MMD2, also known as DM1 and DM2), with the principal goal of preparing for testing of new MMD treatments as they become available.
Each center in the new network has a particular interest and expertise in MMD, and the centers are distributed across the United States to maximize opportunities for participation in studies. Neurologist Charles Thornton at the University of Rochester Medical Center is the network's overall principal investigator.
The researchers in each center will work together to standardize methods of evaluating disease severity and will determine the best ways to assess whether experimental treatments are beneficial.
The new MMD centers are:
"Recent years have seen rapid progress in the development of molecularly targeted therapy for myotonic dystrophy," said Jane Larkindale, MDA's director of translational research.
"Basic research has shown that this disorder involves a disease mechanism known as 'RNA toxicity,' and translational research has shown that this mechanism is unusually responsive to therapeutic intervention. Based on the current pace of therapeutic development, it is reasonable to expect that new therapies will be advanced into clinical trials in the near future, and the new network will ensure that we're prepared for that."
In 2009, MDA established clinical research networks for amyotrophic lateral sclerosis (ALS) and Duchenne muscular dystrophy (DMD) through five MDA/ALS centers and five MDA clinics with special expertise (see Speeding the Course of Clinical Trials). In 2011, the Association partnered with other groups to form a Charcot-Marie-Tooth disease (CMT) network, which continues to receive MDA support (see Providing a Network for Clinical Research in CMT).
To learn more about MMD1 and MMD2, see: