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 
Please join us for a weekend of hope, updates and education at the Patient and Scientific Conference.
Register Today