SEP 24, 2014 6:00 AM PDT

Translational Medicine for Rare Disease: Lessons from Duchenne Muscular Dystrophy

Speaker
  • Executive Advisor to Life Sciences, Cure Duchenne
    Biography

      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. 
       
       

    Abstract

    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.


    Show Resources
    You May Also Like
    SEP 10, 2020 9:00 AM PDT
    C.E. CREDITS
    SEP 10, 2020 9:00 AM PDT
    Date: September 10, 2020 Time: 9:00am (PDT), 12:00pm (EDT) Osmolality testing is relevant throughout the entire bioprocessing workflow. As customers look to refine mAb and gene therapy workf...
    SEP 03, 2020 9:00 AM PDT
    C.E. CREDITS
    SEP 03, 2020 9:00 AM PDT
    DATE: September 3, 2020 TIME: 09:00am PT, 12:00pm ET xxx Learning Objectives: xxx Webinars will be available for unlimited on-demand viewing after live event. LabRoots is approved as a provi...
    C.E. CREDITS
    This drug development program is designed to create a family of broad-spectrum, pan-coronaviral drugs that respectively inhibit multiple key enzymes required for viral replication. By target...
    JUN 17, 2020 1:30 PM PDT
    C.E. CREDITS
    JUN 17, 2020 1:30 PM PDT
    Understanding the complex interplay between a pathogen and the host response is important to developing effective vaccines and therapeutics. The nCounter® Analysis System and GeoMx®...
    NOV 16, 2020 8:00 AM PST
    C.E. CREDITS
    NOV 16, 2020 8:00 AM PST
    Date: November 16, 2020 Time: 8:00am (PST), 11:00am (EST) CRISPR screening has become the prime discovery tool in modern biomedical research and drug discovery. At the same time, most screen...
    OCT 29, 2020 6:00 AM PDT
    C.E. CREDITS
    OCT 29, 2020 6:00 AM PDT
    Date: October 29, 2020 Time: 6:00am (PDT), 9:00am (EDT), Chronic inflammation can occur as a result of a combination of genetic predispositions and environmental factors. Epigenetic modifica...
    Loading Comments...
    Show Resources