Longitudinal assessment of cognitive function in adult- and late-onset DM1 reveals a pattern of cognitive decline that can be modeled as an early-onset and acceleration of normal aging.
In November, Myotonic Dystrophy Foundation (MDF) staff met with the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and the National Institute of Neurological Disorders and Stroke (NINDS) senior leadership and program/policy staff to discuss research opportunities and federal support for myotonic dystrophy (DM). Discussions focused on two areas: the scientific workforce and biomarker and registration endpoint development.
Scientists from a consulting firm with considerable experience in evaluation of patient-reported outcome measures evaluated available PROMs for their potential in DM1 clinical trials.
Lukasz Sznajder, Ph.D., is developing a mouse model for type 2 myotonic dystrophy, a crucial step that is expected to advance understanding of and therapy for this disease.
Ionis Pharmaceuticals recently announced completion of the IONIS-DMPKRx Phase 1/2a clinical trial, and stated that it expects to have analysis of the data collected through that study completed by early January 2017. The study was designed to evaluate the safety and tolerability of multiple doses of an investigational compound, IONIS-DMPKRx, in adult patients with DM1.