Annual Conference

2020 Michael, Marcia, and Christa Parseghian Scientific Conference for Niemann-Pick Type C Research

Basic, translational, and clinical research towards a greater understanding and a cure for Niemann-Pick Type C disease.

This past year, the annual in-person conference in Tucson, AZ was cancelled due to the COVID-19 / novel Coronavirus pandemic. This conference has always been a phenomenal opportunity to gather families, scientists, and clinicians to share advances and exchange ideas related to curing NPC disease, which is why we are deeply disappointed that we were not able to meet together this year.

Because sharing research progress is essential, we developed a new format in place of the Tucson conference. We hosted a series of virtual symposia throughout the month of May and June to provide key research updates from APMRF-funded researchers and invited speakers, clinical trial updates, and other select research topics.

The symposia was hosted on a password protected Zoom platform and included talks and poster presentations.  Details are below.

Virtual Symposium Dates

These symposia were held from 11am to 2pm EST on:

  • Monday, May 11
  • Monday, May 18
  • Tuesday, May 26
  • Monday, June 1
Parseghian Family Parseghian Family

Virtual Symposium Schedule

Sessions included presentations on NPC1 structure and function, autophagy, cholesterol trafficking, lysosomal biology, therapeutics, gene therapy, and biomarkers.

Session #1


Monday, May 11

11:00am - 2:00 pm (Eastern)
Lipids & Organelles


Featured Speakers
Dr. Andrea Ballabio, Scientific Director, TIGEM
Dr. Mike Henne, Assistant Professor, UT Southwestern
Dr. Robert Keenan, Professor, University of Chicago

Session #2

Monday, May 18
10:30am - 2:00 pm (Eastern)
Cholesterol Transport & Signaling


Featured Speakers
Dr. Adrian Salic, Professor, Harvard Medical School
Dr. Bjorn Pedersen, Associate Professor, Aarhus University
Dr. Arun Radhakrishnan, Associate Professor, UT Southwestern
Dr. Judith Storch, Professor, Rutgers

Session #3


Tuesday, May 26

11:00am - 2:00 pm (Eastern)
NPC1 Models & Approaches


Featured Speakers
Dr. Viviana Gradinaru, Professor, CalTech
Dr. Charles Vite, Professor, U Penn
John Pluvinage, Stanford

Session #4


Monday, June 1

11:00am - 2:00 pm (Eastern)
NPC Screening, Drug Development & Clinical Updates


Featured Speakers
Dr. Yiannis Ioannou, Professor, Mount Sinai School of Medicine
Dr. Marc Patterson, Professor, Mayo Clinic
Dr. Elizabeth Berry-Kravis, Professor, Rush U