5 Slides: Gene therapy and the promise for rare disease – State of Reform – State of Reform

Gene therapy represents a new generation of medicine that shows great promise in the fight against rare genetic diseases. The potential long-term transformative benefits could reduce, or even eliminate the ongoing costs of supporting patients and managing diseases. In this conversation, thought leaders will discuss the promise of gene therapy, how policy makers are responding, and what obstacles stand in the way of wide-spread gene therapy treatments.

Date: Thursday, Oct. 21st, 2021

Time: 12:00 1:00pm PDT

Panelists:

Jennifer Hodgeis the US Rare Neurology Medical Team Lead at Pfizer. Over the past 9 years, she has contributed in roles of increasing responsibility across Early Pipeline/Gene Therapy, Sickle Cell Disease, Hemophilia and I&I where she played an important role in the US & EU launches of XELJANZ for Rheumatoid arthritis. She received her PhD in Immunology and completed two Post-Doctoral Fellowships at Harvard Medical School & the Yale University School of Medicine.

Carolina Sommer is the Founder of the Northwest Rare Disease Coalition, Founder of the Seattle Rare Disease Fair, Co-Founder of the ABC Kind Program, and Author of the Lucys Journey books. She is also a member of the Rare Disease Access Working Group with EveryLife Foundation, We Work for Health, Voters for Cures, and the WA Health Access Network.

Ryan Fischer serves as the Chief Advocacy Officer for Parent Project Muscular Dystrophy and has been with the organization for 16 years. Within PPMD, Ryan oversees patient advocacy, patient-focused drug development initiatives including patient-preference research, and the strategic development of the largest patient reported registry in Duchenne developed by PPMD, The Duchenne Registry.

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5 Slides: Gene therapy and the promise for rare disease - State of Reform - State of Reform