As we close out 2025, we are reflecting on a year of unprecedented momentum. This year, we
- Successfully initiated our first-ever 1-patient Clinical Trial, with the participant already having received five doses of an antisense oligonucleotide (ASO) medicine,
- Reached a significant milestone in our Natural History Study by recruiting 35 participants
- Published the first evidence-based Clinical Care Guidelines to empower families and clinicians, and
- Kicked off a global Prevalence Study in partnership with Mass General Brigham to better estimate the number of people affected by ADLD.
View our recap here: https://www.youtube.com/watch?v=itVTgz-Fx3g
These achievements, bolstered by the addition of four new board members and a new research grant, have transitioned us from simply understanding ADLD to actively treating it.
Our progress has set the stage for an even more ambitious 2026. Our primary scientific goals include
- Expanding the 1-patient clinical trial to more participants,
- Exploring and validating additional biomarkers for ADLD, and
- Completing the stand-up of our drug discovery pipeline by testing new approaches in both cell and mouse models
To achieve this, we have set a fundraising goal of $550,000. These funds are critical to sustaining our scientific team, continuing the Natural History Study, and ensuring we have the regulatory support needed to bring new treatments to our community.