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Session 1: Rare Diseases
Session Chair(s)
Philip Gatti, PhD
Pharmacologist
FDA, United States
Edward M. Kaye, MD
Chief Executive Officer
Stoke Therapeutics, United States
Personalized medicine is a major goal in development of new drugs and in medical care in general. Unfortunately, this is very difficult considering the myriad of genetic variations and pathological mutations in people. Oligonucleotide treatments, however, allow for the targeted development of treatment in not only rare and ultrarare diseases, but recently for one person. In today’s Rare Diseases session, the speakers will demonstrate examples of this type of development program with its successes and challenges.
Speaker(s)
Batten Disease Patient Story Update
Timothy W. Yu, MD, PhD
Boston Children’s Hospital, United States
Associate Professor, Harvard Medical School; Division of Genetics and Genomics
Ultra-Rare Disease Development Scenario
Matt R. Buck, JD
Ionis Pharmaceuticals, Inc., United States
Vice President, Regulatory Affairs
Ultra-Rare Disease Development Scenario
Lucas Kempf, MD
Parexel, United States
Vice President, RCS