All times listed are Eastern Standard Times. All activities are located at Arnold J. Capute, MD, MPH Conference Center, 1741 Ashland Avenue, 8th Floor. Baltimore, MD unless otherwise indicated.
Tentative Agenda
Friday, July 26
Clinic and Research visits (by appointment only)
Families Arrive: Check into hotel
LBSL Research Lab Tours
6:30 – 8:00 p.m. Welcome Reception
Location: Residence Inn by Marriott Baltimore at The Johns Hopkins Medical Campus
800 N Wolfe Street, Baltimore MD
Saturday, July 27
*Full breakfast is available at the hotel for overnight guests starting at 6:30 am
8:30 - 9:00 a.m. Child Care Drop off & Continental Breakfast*
9:00 - 9:15 a.m. Welcome
Brad Schlaggar, MD, PhD; President and CEO
Kennedy Krieger Institute
Ali Fatemi, MD; MBA
Chief Medical Officer
Director, Moser Center for Leukodystrophies
Kennedy Krieger Institute
Beth McGinn
Executive Director and Co-Founder
Cure LBSL
Mike McGinn
Co-Founder
Cure LBSL
9:15 – 9:35 a.m. Cure LBSL Advocacy Updates
Melody Kisor, MS
Director of Patient Engagement
Cure LBSL
9:35 – 9:50 a.m. Self-Care -- Yoga for Mitochondrial and Neurological Disorders
Christy Balcells, RN
Yoga Lemonade
Eduardo Balcells, MD
Yoga Lemonade
Julie Wallace
Yoga Lemonade
9:50 – 10:50 a.m. Natural History Study Updates
Amena Smith Fine, MD, PhD
Assistant Professor, Neurology and Developmental Medicine
Kennedy Krieger Institute
10:50 – 11:00 a.m. Break
11:00 – 12 p.m. Infection & Fever
Peter McGuire, MS, MBBCh
National Institutes of Health MINI Study
Eliza Gordon-Lipkin, PhD
National Institutes of Health MINI Study
Sharon Kruk, RN
National Institutes of Health MINI Study
Ali Fatemi, MD, MBA
Kennedy Krieger Institute
Amena Smith Fine, MD, PhD
Kennedy Krieger Institute
Parent/Caregiver Perspectives
12:00 – 1:00 p.m. Lunch and Learn
1:00 - 1:50 p.m. Gene Therapies and Trial Readiness Panel
Michelle Campbell, PhD
Associate Director, Stakeholder Engagement & Clinical Outcomes, Office of Neuroscience
U.S. Food and Drug Administration
Zollie Yavarow, PhD
Post-doctoral Fellow
COMBINEDBrain
Ali Fatemi, MD, MBA
Kennedy Krieger Institute
1:50 – 2:00 p.m. Break
2:00 – 2:50 p.m. Caring for Medically Complex Individuals
Maura Ruzhnikov, MD
Moser Center for Leukodystrophies
Kennedy Krieger Institute
Nancy Yeh, MD
Medical Director, Inpatient Rehabilitation Unit
Kennedy Krieger Institute
Melanie Brown, MD
Medical Director of Pediatric Palliative Care
The Johns Hopkins School of Medicine
Sydney Faw, LMSW
Kennedy Krieger Institute
2:50 – 3:00 p.m. Break
3:00 – 3:50 p.m. Sleep, Energy and Rest
Jennifer Accardo, MD, MSCE
Assistant Professor, Department of Pediatrics
Children’s Hospital of Richmond at VCU
Alexander Sercel, PhD
Director of Scientific Affairs, Mitochondrial Disease Biology
MitoWorld
Ed Blakey, Esq.
Cure LBSL
3:50 – 4:00 p.m. Break
(Families remain at conference; scientists and researchers go off-site for discussion)
4:00 – 4:50 p.m. Physical Therapy and Rehabilitation
Jennifer Keller, MS, PT
Kennedy Krieger Institute
4:50 – 5:15 p.m. Capturing the Patient Voice & Closing Remarks
Melody Kisor
Director of Patient Engagement
Cure LBSL
Beth McGinn
Executive Director and Co-Founder
Cure LBSL
Mike McGinn
Co-Founder
Cure LBSL
Sunday, July 28
Full breakfast at the hotel for overnight guests
8:30 - 9:00 a.m. Child Care Drop and Continental Breakfast
9:15 - 10:30 a.m. Moser Center for Leukodystrophies Research Update:
Gene Therapies for LBSL
Christina Nemeth Mertz, PhD
Assistant Professor of Neurology, Moser Center for Leukodystrophies, Kennedy Krieger Institute
Ines Garofolo, BS
Research Technician, Moser Center for Leukodystrophies
Kennedy Krieger Institute
Bayley Lindsay, BS
Research Technician, Moser Center for Leukodystrophies
Kennedy Krieger Institute
10:30 – 10:45 a.m. Break
10:45 - 11:45 a.m. Biomarkers Panel
Johan Van Hove, MD, PhD
Professor, Pediatrics-Clinical Genetics and Metabolism
University of Colorado School of Medicine
Bela Turk, MD
Postdoctoral Fellow, Johns Hopkins University School of Medicine
11:45 a.m. – 12:00 p.m. Question and Answer Session
12:00 – 1:00 p.m. Lunch and Poster Session
1:00 – 1:30 PM Talk Title TBD
Kathryn Morelli, PhD
Assistant Professor, Department of Neurological Sciences, Larner College of Medicine
University of Vermont
1:30 – 2:15 PM Alternative Functions of DARS2 and its Consequences
Haissai Cui, PhD
Assistant Professor, Department of Chemistry
University of Toronto
2:15 – 2:30 p.m. Question and Answer Session and Break
2:30 – 3:00 p.m. Novel Mouse Models of LBSL
Marc Engelen, MD, PhD
Neurologist and Clinical Researcher
Amsterdam University Medical Centers
3:00 – 3:30 p.m. Nontraditional DARS2 Model Systems
Marni Falk, MD
Executive Director of the Mitochondrial Medicine Frontier Program
Children’s Hospital of Philadelphia.
Cristina Remes, PhD, MSc
Research postdoctoral fellow, Mitochondrial Medicine Program
Children's Hospital of Philadelphia.
3:30 – 3:45 p.m. Question and Answer Session and Break
3:45 – 4:15 p.m. Is LBSL More Prevalent Than We Think?
Carmen Glaze, BS
Variant Coordinator
The Broad Institute of MIT and Harvard
Kathryn Russell, BS
Variant Coordinator
The Broad Institute of MIT and Harvard
4:15 – 4:45 p.m. Drug Repurposing: Turning Old Cures into New Ones
Tyler Beck, PhD
Program Director, Office of Drug Development Partnership Program
National Center for Advancing Translational Sciences (NCATS)
4:45 – 5:15 p.m. Review, Acknowledgements, and Closing remarks
Beth McGinn
Executive Director and Co-Founder
Cure LBSL
Mike McGinn
Co-Founder
Cure LBSL
Ali Fatemi, MD, MBA
Kennedy Krieger Institute
Monday, July 29
Clinic and Research Visits (by appointment only)
Patient Voice Activities
Patient and Family Resource Fair
LBSL Research Lab Tours