Upcoming EveryLife Foundation Scientific Workshop on May 21, 2024 will Address Challenges in Developing Therapies for Ultra-Rare Diseases

May 20, 2024By Sarah Wicks

On May 21, 2024, the EveryLife Foundation for Rare Diseases (ELF) will host a Scientific Workshop at the National Press Club in Washington, D.C. aimed at identifying and characterizing the challenges in developing therapies for ultra-rare diseases and conditions that affect exceedingly small populations.

The workshop will drive in-depth discussions from leaders across federal agencies, the patient advocacy community, industry, academia and policy to examine the current state of drug development for patients with ultra-rare diseases and identify the considerations and implications of establishing a formal statutory definition and framework for “ultra-rare.”

Speakers at the Scientific Workshop will include Dr. Peter Marks, Director of FDA’s Center for Biologics Evaluation and Research, Dr. Janet Woodcock, former Principal Deputy Commissioner of the FDA, Dr. Janet Maynard, Director of the FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicines, and Dr. Joni Rutter, Director of the National Center for Advancing Translational Sciences, and Hyman, Phelps & McNamara’s own Frank Sasinowski who also serves as the Vice Chair of the Board for ELF.

The workshop is free to attend and available to all.  Due to limited spacing, new registrants must attend virtually.  More information on the Scientific Workshop can be found here and registration for the live-stream can be found here.