June 2, 2017

Noon-2pm                     Registration
2:00 – 5:00 pm              Session 1:  NPC1 Function and Lysosomal biology

June 3, 2017
8:00 am                          Breakfast
8:30 am – Noon            Session 2: Cholesterol Trafficking and Lysosomal Biology
Noon – 1:00 pm            Lunch
1:00 pm – 2:30 pm       Session 3: Translational Therapeutics
5:00 pm – 6:30 pm       Poster Session #1
6:30 pm                         Dinner

June 4, 2017   

8:00 am                         Breakfast
8:30 am – Noon           Session 4: Translational Therapeutics, Gene Therapy and Clinical trials, and Panel Discussion
Noon – 1:00 pm           Lunch
1 pm – 2:30 pm           Session 5: Biomarkers and New Born Screening
5:00 pm – 6:30 pm      Poster Session #2
6:30 pm                        Reception and Dinner and Remarks by APMRF

June 5, 2017     

8:00 am                        Breakfast

Presentations

There will be opportunities to submit abstracts for talks and the poster session.  All submissions are due by April 1, 2017.

Speakers include:

Keynote address by Dr. Thijn Brummelkamp, Genetic studies on the function of NPC1 in pathogen entry and cholesterol trafficking

Dr. Roberto Zonco, Mechanisms of cholesterol sensing by the master growth regulator mTORC1 kinase

Dr. Wei Zheng, Therapeutic development for treatment of NPC: beyond cyclodextrin

Dr. Arun Radikrishnan, Intracellular trafficking routes of cholesterol

Dr. Kenji Ohgane, Identification of Chaperone Drugs for NPC through Drug Repurposing Screening

Dr. Yiannis Loannou, Targeting the Mitochondria-ER-Lysosome axis for the treatment of multiple lysosomal storage diseases

Dr. Charles Vite, Gene therapy in the CNS in large animal models

Dr. Xuntian Jiang, Biomarker discovery and development of diagnostics for early detection of NPC disease

Dr. Melissa Wasserstein, Piloting Newborn Screening for Lysosomal Storage Diseases

Dr. Don Bailey, Gathering the Evidence Needed for Newborn Screening

There will be additional presentations selected from the abstracts and from investigators in the NPC community