Summer 2026 Newsletter
Spotted in Boston: LBSL Progress on Display
Dr. Christina Nemeth Mertz of Kennedy Krieger Institute presented on the DARS2-AAV9 gene therapy program at the American Society of Gene and Cell Therapy's Annual Meeting in Boston last month — one of the most important gene therapy conferences in the world. It was a full house for her presentation, and shows that our work is gaining visibility across the scientific community!
Join Us Virtually July 24-26!
The fifth biennial LBSL Patient & Family Conference is coming — and it’ll be the best one yet. Cure LBSL and Kennedy Krieger Institute are proud to co-host three extraordinary days of hope, science and community next month.
Join us virtually as we dig into what's happening in the lab, what's on the horizon and what it means for families living with this ultra-rare disease today. Whether you're a patient, caregiver, family member, researcher, clinician or advocate, this is an LBSL event you don't want to miss! See the draft agenda!
LBSL Tennis & Pickleball Tournament Coming Soon!
Join us for our first LBSL Tennis and Pickleball Tournament on Sunday, Sept. 27 in Reston, Va. It will be an incredible day — joyful, competitive, and full of people who care about this community. You don't want to miss it!
Play: 2-person tennis or pickleball teams
Learn: Sign up for a youth tennis clinic or adult beginner pickleball clinic
Sponsor: Sponsorship levels start at $500!
Can't come? Buy a yard sign with a note of support
Mark Your Calendar!
Sept. 20 is LBSL Awareness Day, and this year, we're planning to make this the biggest September ever for LBSL! But we're going to need your help. We'll share more details next month.
Patient Registry Opportunity
Cure ARS recently launched a patient registry through NORD's IAMRARE platform. (LBSL is caused by one of the 19 ARS genes: DARS2.) Patient registries are a powerful tool — they help researchers understand the full scope of LBSL, identify study participants and make the case for drug development investment.