SEP 24, 2014 06:00 AM PDT

Translational Medicine for Rare Disease: Lessons from Duchenne Muscular Dystrophy

  • Executive Advisor to Life Sciences, Cure Duchenne

      Dr. Ward is an experienced strategist, helping companies large and small transition technology into products. Dr. Ward earned her Bachelors, Masters and PhD degrees in Pharmacology from the University of Manchester, UK. She has over 50 articles in peer-reviewed journals, holds 9 patents, and is a frequent invited speaker.  Dr. Ward is the Independent Director for Provasculon Inc., is a mentor for MBA students at Boston University, and recently completed 6 years as a Trustee, Treasurer and Executive Committee member at the Cambridge School of Weston, a leader in progressive education. Dr Ward has held prominent positions at Sterling Drug, Wyeth Research, and Millennium Pharmaceuticals. Since 2004, Dr. Ward has built a consulting practice crafting the initial R&D strategy for Alnlyam Pharmaceutical, establishing a global program that markedly enhanced quality of development candidates for Novartis (NIBR), and delivering value-based commercialization strategies for software companies focused on Life Sciences including Definiens, Conformia (acquired by Oracle), and GNS Biotech.   Most recently, Dr. Ward has extended her practice to rare disease foundations engaged in sponsorship and venture investment in drug-focused programs and Biotechnology companies. 

    Duchenne Muscular Dystrophy (DMD) is an X-linked genetic disease occurring in 1 in 3,500 births, and is uniformly fatal in boys. It is caused by any one of over 50 known mutations in the gene for dystrophin that result in the production of little to no dystrophin protein a large protein with both structural and signaling capacity that is critical to the normal function of all muscle types. DMD shares many characteristics with other rare diseases that have proven distinctly challenging to drug developers. This presentation will review those issues and illustrate how translational science has, and can, dramatically accelerate the path to potentially life-saving treatments for rare disease patients.

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