Rare Disease Day 2020

Rare Disease Day

Friday, February 28, 2020

Thank you for coming!

Keynote

"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.

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

Rare Disease Day Sponsor Footer

Program/Abstract Booklet

Program/Abstract Booklet

Rare Disease Day Proclamation

Rare Disease Day Proclamation

Rare Disease Advisory Council Progress Report

Rare Disease Advisory Council Progress Report

Patient Advocacy Group Breakfast Presentation

Patient Advocacy Group Breakfast Presentation

News Coverage

News Coverage

Letter from Senator Amy Klobuchar

Letter from Senator Amy Klobuchar

Sponsors

Sponsors

  • 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