Researchers to launch new cohort in DMD natural history study

Funded by Sarepta Therapeutics, the effort will build on previous longitudinal study

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Researchers are planning to enroll a new group of patients in a natural history study to collect data about how Duchenne muscular dystrophy (DMD) progresses and affects a person’s life.

Natural history studies don’t test an experimental therapy, but instead work to collect detailed information on how a disease manifests and progresses without treatment. This type of research can provide a crucial benchmark to measure potential treatments’ effectiveness.

The work, funded by Sarepta Therapeutics, will build on a longitudinal study (NCT00468832) from 2003-2016.

The study included 440 families at 20 centers across nine countries. Data from it have helped researchers better understand how genetic variations affect disease progression and identify biological markers of disease activity. Its findings have also served as a baseline to assess the effectiveness of new DMD treatments.

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“Data from our initial longitudinal study has been used in the development of novel therapies that today are being used by individuals with DMD worldwide,” Erik Henricson, PhD, a professor at the University of California, Davis (UC Davis) and co-principal investigator of the study, said in a university news release.

Henricson said the study’s investigators are “excited to begin enrollment of a new global cohort of patients with DMD.”

“We look forward to continuing this longitudinal study as long as necessary to establish effective long-term treatments for people with DMD,” he said.

The study is being led by scientists at UC Davis in collaboration with an international group of neuromuscular disease investigators called the Cooperative International Neuromuscular Research Group (CINRG) and the contract research organization Therapeutic Research in Neuromuscular Disorder Solutions (TRiNDS).

“UC Davis has an established track record of fostering partnerships between academic and industry investigators in noncompetitive areas of science. These collaborative scientific programs can benefit the global research community through novel discoveries that can lead to life-changing medical innovations,” Henricson said. “Through the extraordinary efforts of our longtime colleagues from CINRG, the UC Davis Duchenne Natural History Study has grown over the past 20 years to become a model of collaborative efforts and is a worldwide data resource for academia, patient advocacy groups, industry, and government agencies alike.”