2017 Scientific Conference:
Development of Therapies for Peroxisome Biogenesis Disorders
The 2017 Scientific Conference was held at the Embassy Suites at the Chevy Chase Pavilion in Washington, D.C. on July 13-14, 2017.
Scientific Conference Agenda
All sessions take place in the Chevy Chase Ballroom in the Embassy Suites Conference Center unless otherwise indicated.
Wednesday, July 12
5:00 – 7:30 p.m. Embassy Suites Managers’ Reception and Conference Pre-Registration Willie’s Bar
Thursday, July 13
7:00 – 8:30 a.m. Breakfast and Registration Atrio Café
8:30 – 9:30 a.m. Welcome and what are we here for?
Joseph Hacia (USC), Nancy Braverman (McGill University), Melissa Bryce Gamble (GFPD)
9:30 a.m. – 12:00 p.m. Session 1: Overview for peroxisome biogenesis disorders and model systems
Moderator: Gerald Raymond (University of Minnesota)
9:30 – 10:15 a.m. Clinical overview of PBD-ZSD and RCDP
Bwee Tien Poll-The (Video Available Here) and Femke Flower (University of Amsterdam)
10:15 – 10:45 a.m. Natural history study for PBDs
Nancy Braverman (McGill University) (Presentation available here) (Video Available Here)
10:45 – 11:15 a.m. The PBD-ZSD metabolome
Michael Wangler (Baylor University College of Medicine) (Presentation Available here)
11:15 – 12:00 p.m. Vertebrate and invertebrate models of PBDs
Myriam Baes (University of Leuvan, Belgium) (Presentation available here)
12:00 – 1:00 p.m. Lunch (mentor/trainee mixer) Atrio Café
1:00 – 3:00 p.m. Session 2: Small molecule therapies targeting peroxisome assembly
Moderator: James Inglese (NIH/NCATS)
1:00 – 1:30 p.m. Rare disease therapy development at NCATS
Chris Austin (NIH/NCATS) (Presentation available here)
1:30 – 2:00 p.m. Betaine and flavonoid therapies for PBD-ZSD
Nancy Braverman (McGill University) (Presentation available here) (Video available here)
2:00 – 2:30 p.m. Screening for small molecules that improve peroxisome assembly
Joseph Hacia (USC) (Presentation available here)
2:30 – 3:00 p.m. Panel Discussion/Break
3:00 – 5:00 p.m. Session 3: Drug therapies targeting neurological aspects of disease
Moderator: Ali Fatemi (Kennedy Krieger Institute)
3:00 – 3:30 p.m. Overview of neurological aspects of PBDs
Genevieve Bernard (McGill University)
3:30 – 4:00 p.m. Use of neurosteroids for the treatment of pediatric refractory seizures
Mark Wainwright (Northwestern University, Feinberg School of Medicine) (Presentation Available here)
4:00 – 4:30 p.m. Small molecule therapies for inherited intellectual disabilities
Jonathan Picker (Boston Children's Hospital) (Presentation Available Here)
4:30 – 5:00 p.m. Drug-based modulation of endogenous stem cells to promote functional remyelination
Paul Tesar (Case Western Reserve School of Medicine)
5:00 – 5:15 p.m. Concluding remarks for the day, Joseph Hacia (USC), Nancy Braverman (McGill University)
5:15 – 7:00 p.m. Cocktail Hour, Dinner and Poster Session (mentor/trainee mixer) Maggiano’s Little Italy
7:00 – 7:30 p.m. Family perspectives, Mindy Lee (RhizoKids International), Melissa Bryce Gamble (GFPD) (Presentation Available Here)
7:30 – 8:30 p.m. Keynote: Therapeutic Development for Rare Diseases: Challenges and Opportunities Francis S. Collins (NIH)
Friday July 14
7:00 – 7:50 a.m. Breakfast Atrio Café
7:50 – 8:00 a.m. Opening Remarks
8:00 – 9:45 a.m Session 4: Biochemical approaches to therapy
Moderators: Paul Watkins and Ann Moser (Kennedy Krieger Institute)
8:00 – 8:30 a.m. Bile acid replacement therapy for PBD-ZSD, Bwee Tien Poll-The (University of Amsterdam)
8:30 – 9:00 a.m. Plasmalogen replacement therapies Pedro Brites (IMDC, Porto, Portugal)
9:00 – 9:30 a.m. Clinical Trial: Strategies for documenting RCDP natural history Michael Bober (Alfred I. DuPont Hospital for Children)
9:30 – 9:45 a.m. Panel Discussion/Break
9:45– 11:30 a.m. Session 5: Therapies for vision and hearing loss Moderator: Nancy Braverman (McGill University)
9:45 – 10:15 a.m. Cell-based therapies for retinal degenerative diseases Kapil Bharti (NIH/NEI)
10:15 – 10:45 a.m. Retinal gene therapy for PBD-ZSD, Jean Bennett (University of Pennsylvania)
10:45 - 11:15 a.m. Gene therapy for hearing loss, Jeffrey Holt (Boston Children’s Hospital)
11:30 a.m. – 1:00 p.m. Working Lunch: Planning Breakout Sessions Atrio Café Moderators: Hacia, Braverman, Rizzo, Raymond and Bose
1:00 – 2:30 p.m. Session 6: Gene and stem cell therapy for CNS disorders Moderator: William Rizzo (University of Nebraska)
1:00 – 1:30 p.m. Gene therapy for giant axonal neuropathy Steven Gray (University of North Carolina)
1:30 – 2:00 p.m. Gene therapy for adrenomyeloneuropathy, Florian Eichler (Massachusetts General Hospital) (Presentation available here)
2:00 – 2:30 p.m. Stem cells in the treatment of neurodegenerative disorders Eva Feldman (University of Michigan)
2:30 – 3:30 p.m. Session 7: Therapies for blood and liver disorders, Moderator: Gerald Raymond (University of Minnesota)
2:30 – 3:00 p.m. Hematopoiesis and PBD-ZSD, Troy Lund (University of Minnesota)
3:00 – 3:30 p.m. Gene therapy for liver disorders, Katherine High (Spark Therapeutics)
3:45 – 5:15 p.m. Break Out Sessions: Towards treatment, Moderators: Hacia, Braverman, Rizzo, Raymond, Bober, Bose.
5:30 – 7:30 p.m. Dinner with the GFPD Family Conference Participants Maggiano’s Little Italy
7:30 – 8:45 p.m. Presentations by small group session leaders to all conference participants
8:45 – 9:00 p.m. Summary Charge and Closing, Joseph Hacia (USC) and Nancy Braverman (McGill University)
