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08/14/2018 - 8:31am

A key driving factor behind DM1 is the instability of expanded CTG repeats in DMPK, resulting in both germline and somatic expansions in repeat length. Variant CCG-containing CTG repeats are more stable and yield a milder phenotype than corresponding “pure” DM1 alleles.

08/09/2018 - 5:07pm

One of the challenges faced by doctors treating patients with myotonic dystrophy type 1 (DM1)—and drug developers designing clinical trials—is the broad difference in the way the disease manifests itself and progresses from patient to patient. MDF Scientific Advisory Committee member Dr. Guillaume Bassez, a neurologist at the Institut de Myologie in Paris, has identified subgroups of the DM population to help address this issue.

07/17/2018 - 8:51am

MDF strongly encourages patients, families, clinicians, and researchers to respond to the NIH’s Request for Information on the Wellstone Center Program.

07/17/2018 - 8:46am

A new U.S. National Academy of Sciences (NAS) report—Returning Individual Research Results to Participants: Guidance for a New Research Paradigm—should be read by all clinical researchers.

07/17/2018 - 8:45am

The OPTIMISTIC clinical evaluation of exercise training and cognitive behavioral therapy paradigms comes to publication.


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