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

8:30amRegistration BeginsSpeaker/Participants
10:00amWelcome RemarksSean Kassen, PhD,
Director, Ara Parseghian Medical Research Fund
10:10amMeeting Agenda and Desired OutcomesTimothy R. Franson, MD,
Moderator
10:20amFDA’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
10:35amOverview of Niemann-Pick Type C Disease and Current TreatmentsMarc C. Patterson, MD,
Professor of Neurology, Pediatrics and Medical Genetics, Mayo Clinic Children’s Center
10:55amPanel Format Description & Demographics PollTimothy R. Franson, MD,
Moderator
11:00amPanel on Discussion Topic 1: Symptoms of Niemann-Pick Type C disease (late infantile and juvenile forms) and daily impacts that matter mostPanel of Niemann-Pick Type C patients and patient representatives
11:30amFacilitated Discussion: Topic 1Patients and patient representatives in the audience
12:05pmLunch
1:00pmAfternoon WelcomeTimothy R. Franson, MD,
Moderator
1:05pmPanel 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
1:40pmFacilitated Discussion: Topic 2Patients and patient representatives in the audience
2:20pmPanel on Discussion Topic 3: Symptoms of Niemann-Pick Type C disease (early infantile and adult forms) and daily impacts that matter mostPanel of Niemann-Pick Type C patients and patient representatives
2:50pmFacilitated Discussion: Topic 3Patients and patient representatives in the audience
3:30pmOpen Public Comment
3:50pmKey Points from Today’s DiscussionTimothy R. Franson, MD,
Moderator
3:55pmClosing RemarksSean Kassen, PhD,
Director, Ara Parseghian Medical Research Fund
*Please not that the agenda is subject to change