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Patients as Partners in Ultragenyx LC-FAOD Research

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Join Energy in Action host, Stephanie Tomlinson as she interviews Emil Kakkis and Jessica Riviere from Ultragenyx Pharmaceutical.

On this podcast, Ultragenyx will share how the company entered into LC-FAOD research, its research efforts, and how LC-FAOD patient voices and community engagement are central to the planning, implementation, and advancement of Ultragenyx research and programs. Ultragenyx is proud to partner with MitoAction to provide the LC-FAOD community with information and updates through a series of discussions on MitoAction’s Energy in Action podcast and Monthly Expert Webinar Series.

The podcast will be available on our website, and Spotify, Google Podcasts, Apple Podcast and Amazon Music podcast libraries.

*This podcast episode is intended for U.S. members of the MitoAction community.*

About the Speaker

Dr. Emil D. Kakkis

Dr. Emil D. Kakkis, M.D., Ph.D. is Chief Executive Officer, President and Founder of Ultragenyx Pharmaceutical. Over the last 25 years, Dr. Kakkis is best known for his work developing novel treatments for rare diseases and advancing the cause of rare disease treatment through advocacy on policy issues by founding and supporting the EveryLife Foundation for Rare Diseases. In 2019, he received BIO’s Henri Termeer Visionary Leadership award for this work and a Lifetime Achievement Award from the National MPS Society.

Dr. Kakkis began his academic research at Harbor-UCLA developing an enzyme replacement therapy for the rare disorder MPS I. After joining BioMarin in 1998, he guided the development and approval of two more treatments for rare diseases, MPS VI and PKU and has contributed to the development of approved or development stage products of four other rare diseases (CLN2, MPS-IVA, PKU, Achondroplasia).

Dr. Kakkis went on to found Ultragenyx in 2010 to focus on developing as many rare and ultra- rare disease therapeutics as possible. The company went public in January 2014 (NASDAQ: RARE) and has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. Since its founding, Ultragenyx has received approvals for XLH, TIO, MPS VII, and LC-FAOD.

Dr. Kakkis graduated from Pomona College, magna cum laude and received combined M.D. and Ph.D. degrees from the UCLA Medical Scientist Program. He completed both a Pediatrics residency and Medical Genetics Training Fellowship at Harbor-UCLA Medical Center.

Jessica Riviere

Jessica Riviere, MHS leads Global Patient Advocacy and Patient Engagement at Ultragenyx, where she and her passionate team forge strategic alliances with diverse patient advocacy groups to understand patient and caregiver perspectives across the drug discovery continuum.  Her passion is building capacity that enables groups to be inform drug discovery, drive disease awareness campaigns, support equitable access to care and treatment and co-create meaningful support services for people impacted by disease and their caregivers.

Early in Jessica’s career she worked on advancing the Clinton Healthcare Plan as a legislative aide to U.S. Senator Edward Kennedy.  It was during her tenure at Kennedy’s office that Jessica came to appreciate the power of communities to make change and impact public policy for the greater good.

Jessica’s advocacy experience spans multiple rare diseases, diabetes, HIV/AIDS and immunology with several companies including Biogen, AbbVie, Bristol Myers Squibb and Novo Nordisk.

Jessica holds a BA in Psychobiology from Mount Holyoke College and a Masters of Health Science in Health Policy from the John Hopkins Bloomberg School of Public Health.

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