Subcellular compartmentalization of MBNL1 plays a key role in regulation of neurite differentiation and its disruption may contribute to CNS defects in DM.
The National Human Genome Research Institute has issued new funding opportunities for research targeted using a patient’s genomic information to inform clinical care.
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 manifest itself and progresses from patient to patient. Dr. Guillaume Bassez, at the Institut de Myologie in Paris, has identified subgroups of the DM population to help address this issue.
When the 11th meeting of the International Myotonic Dystrophy Consortium (IDMC-11) met in San Francisco in September 2017, Dr. Giovanni Meola was in attendance, as he has been since the first biennial meeting in Paris, France, in 1997.
Close monitoring of respiratory function reduces respiratory complications in DM1.