Join patients, families, advocates, researchers, clinicians, and regulators from around the world at this exciting event. You can attend in-person in Baltimore, Maryland, or virtually on Zoom. This event will be recorded and shared. All times are stated in Eastern Daylight Time (UTC-5).
Tentative Agenda
Friday, July 26
- Kennedy Krieger Institute clinic visits (by appointment)
- Research visits (by appointment)
- Capturing the patient voice
- Welcome reception
- LBSL art gallery
Saturday, July 27 - Living With LBSL
- Childcare drop off
- Breakfast
- Welcome from Kennedy Krieger Institute and Cure LBSL
- LBSL advocacy updates
- Yoga for Mitochondrial & Neurological Disorders
- Research & Natural History updates
- Physical Therapy and Rehabilitation
- Lunch
- Caring for Medically Complex Individuals
- School & Academic Support
- Nutrition, Supplements, & Digestive Health
- Sleep, Energy, & Self Care
- Q & A - Ask the Clinician
- Closing remarks
Sunday, July 28 - Searching For a Cure
- Childcare drop off
- Breakfast
- Moser Center for Leukodystrophies Research Update: Gene Therapies for LBSL
- LBSL International: Collaboration around the World
- Lunch
- Nontraditional DARS2 model systems
- Alternative functions of DARS2 and its consequences
- Gene Therapies and Trial Readiness Panel
- Is LBSL more prevalent than we think?
- Drug repurposing: turning old cures into new ones
- Closing remarks
Monday, July 29
- Kennedy Krieger Institute clinic visits (by appointment)
- Research visits (by appointment) \
- Lunch voucher for clinic / research participants