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) 

                                              (Presentation available here)

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)


2017 Scientific Conference: Development of Therapie for Peroxisome Biogenesis Disorders