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Wyck Foundation and MDF Grant Awards

In partnership with MDF, the Wyck Foundation awarded a number of new DM research grants.

Toward Effective Biomarkers for DM

Biomarkers of various Contexts of Use are essential for drug development in DM—recent guidance documents and publications point to exciting new opportunities.

Cognitive Changes in Adult DM1: An Acceleration of Normal Aging?

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.

MDF Staff Meets with NIAMS and NINDS

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.

Using PROMS to Evaluate Potential Therapies

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.

Research Fellow Profile: Lukasz Sznajder

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.

Update: IONIS-DMPKRx Phase 1/2a Study

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.

Compliance with Prescribed Medication Among Patients Living with Myotonic Dystrophy

In a recent study by Dr. Richard Moxley, III, MD, and colleagues at the University of Rochester, researchers assessed disease manifestations and adherence to medications for DM1 and DM2 patients. The study was motivated in part by the fact that DM patients need to take multiple prescriptions to manage disease symptoms associated with a number of different body systems.

Research Fellow Profile: Dr. Laura Renna

Laura Renna, Ph.D., thinks there may be more to the insulin resistance story in myotonic dystrophy than abnormal insulin receptor splicing. She intends to probe the question by testing three insulin mimetics in cells taken from DM patients and age-matched controls.

Research Fellow Profile: Dr. Ginny Morriss

Ginny Morriss, Ph.D., is exploring whether reducing levels of the CELF1 protein, which are abnormally high in DM1-affected skeletal muscles, has a positive effect on these muscles. She’ll be studying mice in which disease-causing repeat expansions can be induced at any age.

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