Negotiating the Challenges of Therapeutics Discovery for Rare Diseases

The goals of the Rare Diseases Symposium at UCSF are:

  • To create a community of academic scientists, business leaders, regulatory experts and patient advocacy groups with a common interest to tackle rare diseases

  • To examine the key issues in development of treatments for rare diseases

  • To draw attention to and encourage research in origins and treatments of rare diseases

  • To showcase ground-breaking research in rare diseases at UCSF

  • To foster collaborations between academia, industry, and non-profit organizations 


Draft agenda:

Keynote: Patient Foundation

Sponsors panel: How does Rare Disease Research fit into Company strategies/ approaches? 

  • Roche/Genentech
  • Sanofi/Genzyme
  • Ultragenyx 
  • BioMarin

Rare Disease Discovery: Presentation by Emil Kakkis

Role of Disease Foundations: Importance of patient registries

Transformative Cellular Technologies in Rare Disease Research 

Academic & Industry Case-Studies

Challenges of Rare Disease Development & Clinical Research in Small Patient Populations

  • Presentation & Panel Discussion

Patient & Parent Perspectives

Networking Reception



Friday, March 3, 2017 at 8:30am to 6:30pm

Genentech Hall, Byers Auditorium
600 16th St, San Francisco, CA 94158

Event Type

Lecture/Seminar, Academic


UCSF Medical Center, UCSF Benioff Children's Hospitals, School of Medicine, School of Dentistry, School of Pharmacy, School of Nursing, Graduate Division, Global Health Sciences


Students, Postdocs, Faculty, Staff


Mission Bay



Free for UCSF Faculty/staff/student, $25 for non-UCSF academics & non-profits, $250 for Industry

Clinical & Translational Science Institute (CTSI)


Contact Info


Event Registration Required

This event requires registration.