Duchenne ‘Consumer Advocates’ Have Say in Federal Research Funding for DMD

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by Mary Chapman |

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Duchenne advocates

People known as “consumer advocates” have joined top scientists since 2011 in helping to determine how funds appropriated by the U.S. Congress will be spent on Duchenne muscular dystrophy (DMD) research.

Recently, Tiffany Cook and Clint M. Porter got their turn at patient representation.

Cook and Porter’s influence derived from their role as evaluators of research applications submitted to the U.S. Department of Defense’s Duchenne Muscular Dystrophy Research Program (DMDRP), part of the Congressionally Directed Medical Research Programs (CDMRP).

The DMDRP supports the discovery, development and clinical testing of Duchenne therapies, devices, and other inventions.

As consumer reviewers, Cook and Porter had the opportunity to voice their opinions during scientific peer review sessions last year on the role of research in critical issues, including diagnosis, treatment, and quality of life. And while they represented the patient community, the two also had personal stakes.

“My family has a vested interest in advocating for Duchenne research. Our son was diagnosed in 2017,” said Porter in a press release. He and Cook were nominated for participation by a California branch of CureDuchenne, the global nonprofit focusing on DMD research and patient care.

“The review process was very encouraging, and the scientific panel was very interested in the consumer reviewers’ comments,” Cook added. “Organizations like CureDuchenne, and research funding through CDMRP, provide us hope.”

Representing the Duchenne community is “an honor,” he added, and “a valued opportunity to remind the voting members of the heavy burden families carry and the utmost urgency to develop a cure for all individuals affected by Duchenne.”

Their positions gave them full voting rights during official meetings to decide how the $3.2 million appropriation to the DMDRP in 2019 would best support studies in DMD.

“Consumer advocates are an integral part of the CDMRP’s scientific review process,” said Col. Stephen J. Dalal, CDMRP director. “They provide a key ingredient to the review process — the patient’s perspective — which is real and urgent. The collaboration of consumer advocates alongside the scientists’ subject matter expertise is a truly unique collaboration that is difficult to find in most medical research programs.”

Since 1992, the CDMRP has sought to foster novel approaches to biomedical research in response to needs expressed by the public, the military, and congressional leaders.

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