CureDuchenne Seeds Collaborative Project to Analyze the Natural History of Duchenne Muscular Dystrophy

NEWPORT BEACH, Calif., January 13, 2015 – CureDuchenne announced today that the organization is funding Dr. Susan J. Ward, a consultant to BioPharma, in partnership with Dr. James Signorovitch, Vice President at Analysis Group Inc., on a collaborative project to further the understanding of the natural history of Duchenne muscular dystrophy.

CureDuchenne’s goal is to get life-saving treatments to Duchenne patients as quickly as possible. “Three companies are seeking FDA approval for Duchenne drugs next year, and others will be designing pivotal clinical trials,” said Debra Miller, Founder and CEO of CureDuchenne. “The intent of this collaboration is to give researchers an important new tool to develop better understanding of the natural history of Duchenne, thus providing biotech and Pharma companies new insights to interpret their own data in the future.” 

A significant challenge in developing drugs for the treatment of Duchenne is that the disease naturally progresses at different rates in different patients. “Because functional decline over 12 months can vary greatly between patients, the interpretation of clinical trials may be clouded,” said Ward.

“The project will analyze existing clinical databases with the objective of identifying and classifying trajectories of disease progression of Duchenne patients over time,” said Signorovitch. “Classifying these trajectories into clusters would be helpful for designing clinical trials and understanding drug treatment effects.”

Over the past year, CureDuchenne provided seed funding, expertise and resources to help initiate and establish the project. “We expect that a broad consortium of drug companies, academic institutions and other Duchenne muscular dystrophy organizations will come together to help advance and accelerate this project,” said Miller. “This has the potential to benefit the entire Duchenne community.” 

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About CureDuchenne

CureDuchenne is a national nonprofit organization located in Newport Beach, Calif., dedicated to finding a cure for Duchenne, the most common and most lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys worldwide.

CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy. With the help of CureDuchenne’s distinguished international panel of Scientific Advisors, funds raised by CureDuchenne support the most promising research aimed at treating and curing Duchenne. To date, seven CureDuchenne-funded research projects have advanced into human clinical trials – few health-related nonprofits have been as successful in being a catalyst for accelerating human clinical trials.

About Susan J. Ward, Ph.D.

Dr. Ward brings over 20 years of executive experience in drug discovery and development to challenges that demand creative leadership and inter-disciplinary solutions. She was the first woman in the UK to head a research site in Pharma, oversaw Wyeth’s development and regulatory approvals for key therapeutic advances including Rapamune, Enbrel, Protonix i.v. and the first toxin conjugated antibody, and has held executive corporate roles and consulting relationships with leading Biotechnology companies including Millennium Pharmaceutical, Infinity Pharmaceutical, Alnylam, and GNS Biotech. Dr. Ward serves as the independent Director for emerging Biotech companies, and as a business advisor to life sciences technology companies.

About Analysis Group Inc.

With more than 500 professionals with advanced degrees and expertise in health outcomes research, epidemiology, strategy, biostatistics, economics, and other quantitative disciplines, Analysis Group has established a leadership role in the science, economics, and business strategy of the global health care industry. The firm’s offices are located in Boston, Chicago, Dallas, Denver, Los Angeles, Menlo Park, New York, San Francisco, and Washington, DC; and internationally in Montreal and Beijing.

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