International study on Duchenne muscular dystrophy lays the groundwork for future research

(SACREMENTO)

Researchers from the Department of Physical Medicine and Rehabilitation at the University of California, Davis are launching an international study into the natural history of Duchenne muscular dystrophy (DMD). They will partner with the Cooperative International Neuromuscular Research Group (CINRG) and Therapeutic Research in Neuromuscular Disorder Solutions (TRiNDS), an association of medical and scientific researchers who work to improve the lives of people with neuromuscular disease.

Duchenne muscular dystrophy (DMD) is a progressive disease that mainly affects boys. It is present from birth and causes muscle weakness and loss of muscle function. People with DMD may lose the ability to walk during adolescence. DMD also affects heart, lung, and bone health.

The study, known as the Duchenne Natural History Study, will collect information about the course of the disease in people with DMD receiving standard care in clinics around the world. The aim is to study the progression of the disease as patients age and across different settings in order to improve understanding. These findings will help lay the foundation for future treatment research.

Sarepta Therapeutics is funding the study.

“UC Davis has a track record of building partnerships between academic and industry researchers in non-competitive areas of science,” said Eric K. Henrickson, research co-director and assistant professor in the Department of Physical Medicine and Rehabilitation (PM&R). “These collaborative science programs have the potential to benefit the global research community through new discoveries that could lead to revolutionary medical innovations.”

The two men stand side by side at the top of the outer stairs.
Co-leaders Craig McDonald (right) and Eric K. Henrickson (left).

Building on current research into Duchenne muscular dystrophy

The new project builds on a previous longitudinal study by UC Davis scientists and CINRG staff around the world. This study was conducted from 2003 to 2016 and included 20 institutions from nine countries. It included 440 families. The researchers studied patterns of changes in physical abilities, the severity of medical problems, and how patients sought medical care. The research team also discovered biomarkers associated with disease progression and determined how common genetic variations can alter the pattern and rate of progression of DMD.

“The data from our original longitudinal study were used to develop new treatments that are now being used by people with DMD around the world,” Henrickson explained. “Together with our colleagues at CINRG, UC Davis is pleased to begin enrolling a new global DMD patient cohort.”

Eric Henrikson

The data from our original longitudinal study were used to develop new therapies that are now being used by people with DMD around the world. Together with our colleagues at CINRG, UC Davis is pleased to begin enrolling a new global cohort of patients with DMD.”Eric K. Henrikson, Associate Professor, Department of Physical Medicine and Rehabilitation

Joining Henrikson as study co-leader was Craig McDonald, professor and chairman of PM&R. McDonald is also the lead investigator of a clinical trial of systemic cellular therapy for DMD, which has shown great promise in halting the deterioration of upper limb and heart function in patients.

“Through the incredible efforts of our longtime colleagues at CINRG, UC Davis Natural History Research has grown over the past 20 years into a collaborative effort model and is a worldwide data resource for academia, patient advocacy groups, industry. and government agencies,” Henrikson said. “We look forward to continuing this longitudinal study for as long as necessary to develop effective long-term treatments for people with DMD.”

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