Rare Disease Day 2020

Banner stating "Rare Disease Day"

Friday, February 28, 2020

Two women sitting on a stage in front of an audience
Two women looking and pointing at a board
three women posing behind a display
three women posing behind a display
Two women looking at a display
Man looks at display while another man stands behind it
wide shot of many people sitting at circle tables
two people smiling and reading a display
person points at display and talks while another person looks at the display and listens
wide shot of many displays and people standing next to them
Two people standing behind a display
A woman standing in front of a display board
Two women smiling at the camera
Five people in formal wear standing and smiling at the camera
Two people smiling while pulling their thumbs to their arms with their other hand


"The Rare Disease Treatment Approval Process: Balancing Gold Standard Evidence with Patient-Centered Flexibility"
Sarah Wicks, JD, MPH

Sarah Wicks is an associate at Hyman, Phelps & McNamara, P.C. where she works with sponsors to bring life-changing therapies to market, particularly for persons with rare disease. She is a graduate of the University of Maryland School of Law and Johns Hopkins School of Public Health. Through her efforts, Sarah has gained a growing appreciation for the value of patients’ experiences and the need for the patient voice to inform product development. She is dedicated to furthering the involvement of patients in rare disease drug development as well as advancing and accelerating the development of new breakthrough therapies for those with rare conditions.

Download the PowerPoint

Congratulations to our Research Award Winners!

  1. Abhishek Sathe, Experimental and Clinical Pharmacology graduate student, Underdosing of Benzodiazepines in Patients with Status Epilepticus Enrolled in the Established Status Epilepticus Treatment Trial
  2. Julia Reidl, Medical Scientist Training Program graduate student, Dermal MSCs as a Treatment for Recessive Dystrophic Epidermolysis Bullosa  
  3. Collin Gradin, 3rd year Medical School student, The Importance of Prompt Recognition of Neuromyelitis Optica Spectrum Disorder: A Case Report
  4. Timothy Gauntner, MD, Internal Medicine Residency Program, Hirschsprung Disease in an Infant with L1 Syndrome: Report of a New Case and a Novel L1CAM Variant

Expand all

Program/Abstract Booklet

Rare Disease Day Proclamation

Rare Disease Advisory Council Progress Report

Patient Advocacy Group Breakfast Presentation

News Coverage

Letter From Senator Amy Klobuchar


  • AveXis
  • College of Pharmacy
  • Fairview Pharmacy Services
  • Gillette Children's Speciality Healthcare
  • Greenwich Biosciences
  • Horizon Therapeutics
  • Ligand
  • Orchard Therapeutics
  • Pfizer
  • Prism Research
  • Stem Cell Institute
  • Takeda Pharmaceuticals
  • Upsher-Smith
  • Zogenix
Partners logo