Monday, March 18, 2019
10:00 a.m. – 4:00 p.m.
College Park Marriott Hotel & Conference Center
Meeting Room 2100
Hyattsville, MD

8:30am	Registration Begins	Speaker/Participants
10:00am	Welcome Remarks	Sean Kassen, PhD, Director, Ara Parseghian Medical Research Fund
10:10am	Meeting Agenda and Desired Outcomes	Timothy R. Franson, MD, Moderator
10:20am	FDA’s Approach to Rare Disease Drug Development and the Importance of the Patient Voice	Dragos Roman, MD, Associate Director, Division of Gastroenterology and Inborn Errors Products, Food and Drug Administration
10:35am	Overview of Niemann-Pick Type C Disease and Current Treatments	Marc C. Patterson, MD, Professor of Neurology, Pediatrics and Medical Genetics, Mayo Clinic Children’s Center
10:55am	Panel Format Description & Demographics Poll	Timothy R. Franson, MD, Moderator
11:00am	Panel on Discussion Topic 1: Symptoms of Niemann-Pick Type C disease (late infantile and juvenile forms) and daily impacts that matter most	Panel of Niemann-Pick Type C patients and patient representatives
11:30am	Facilitated Discussion: Topic 1	Patients and patient representatives in the audience
12:05pm	Lunch
1:00pm	Afternoon Welcome	Timothy R. Franson, MD, Moderator
1:05pm	Panel on Discussion Topic 2: Current approach to treating Niemann-Pick Type C (late infantile and juvenile forms)	Panel of Niemann-Pick Type C patients and patient representatives
1:40pm	Facilitated Discussion: Topic 2	Patients and patient representatives in the audience
2:20pm	Panel on Discussion Topic 3: Symptoms of Niemann-Pick Type C disease (early infantile and adult forms) and daily impacts that matter most	Panel of Niemann-Pick Type C patients and patient representatives
2:50pm	Facilitated Discussion: Topic 3	Patients and patient representatives in the audience
3:30pm	Open Public Comment
3:50pm	Key Points from Today’s Discussion	Timothy R. Franson, MD, Moderator
3:55pm	Closing Remarks	Sean Kassen, PhD, Director, Ara Parseghian Medical Research Fund

*Please not that the agenda is subject to change