TRNDS is a day-long symposium on emerging novel therapeutic technologies that are creating new and rapidly expanding treatment options for patients with rare neurological disorders.
Learn the latest research demonstrating breakthrough health technologies for rare neurological disorders.
Discover new ideas and innovations driving therapy development, clinical trials and clinical research.
Join a community of experts dedicated to accelerating the discovery of new therapies for rare neurological disorders.
TRNDS 2019 speakers include leading clinical researchers, forward-thinking health technology leaders, influential patient advocates, and experts in regulatory science. Confirmed speakers include:
Derek Bowen VP Business Development & Strategy, Blackfynn
PJ Brooks Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences, NIH
Wilson Bryan Director, Office of Tissues & Advanced Therapies, Center for Biologics Evaluation & Research, FDA
Tanya Fischer Global Project Head, Sanofi-Genzyme
Jayne Gershkowitz Chief Patient Advocate, Amicus Therapeutics
David Jacoby VP, Head of Clinical Science, BioMarin Pharmaceuticals
Rachel McMinn President & CEO, Neurogene, Inc.
Nancy Yu Co-Founder & CEO, RDMD
Timothy Yu Asst. Prof., Division of Genetics & Genomics, Harvard Medical School
Dina Zand Medical Officer, Center for Drug Evaluation & Research, FDA
TRNDS 2019 program will focus on emerging gene therapy approaches, a new era in enzyme replacement therapy, the future of newborn screening, and novel tools and technologies contributing to the modernization of clinical research. Topics include:
Emerging approaches to treating serious, rare neurological conditions
Personalized medicine in a neurological disease framework
Development of paths to early diagnosis
Use of novel technologies to facilitate high quality, efficient clinical research studies
Ethical considerations in the modernization of clinical trials and development of new therapies
Regional and national leaders in rare diseases, health technologies and clinical trials
Erika F. Augustine
Director of Information Technology, National Organization for Rare Disorders
Craig H. Lipset
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