We’re delighted to share that the first half of 2023 has been productive, and the important work of building partnerships with researchers and clinicians is opening up exciting new avenues of research with potential future impact on the treatment of Autosomal Dominant Leukodystrophy (ADLD). Some of those highlights are included below.
Thank you for your support because none of this would’ve been possible without you. We hope you will remain engaged with the ADLD Center and continue to champion our work toward finding a cure for ADLD. Please consider donating today to support this important work.
Mid-year highlights:
Pilot Research Grants in partnership with the Orphan Disease Center at the University of Pennsylvania: The ADLD Center has awarded three $50,000 pilot research grants in Q2 2023 to stimulate innovative scientific and therapeutic advancements for ADLD. The following researchers were selected:
- Quasar Padiath, University of Pittsburgh, PA USA: The grant will help Dr. Padiath understand which specific types of cells and pathways are implicated in ADLD in order to reveal potential opportunities for targeted treatments.
- Stefano Ratti, University of Bologna, Italy: The grant will help Dr. Ratti develop and study models of ADLD in 3D tissue to understand how ADLD is connected with demyelination in order to reveal potential opportunities for targeted treatments.
- Margot Cousin, Mayo Clinic, MN USA: The grant will help Dr. Cousin set up a clinical trial program for ADLD patients so that when a potential drug is available, the Mayo Clinic will be able to oversee its administration and track its effects over time.
We wrote an ADLD Handbook! The handbook aims to be a comprehensive guide that provides essential information on ADLD, its impact on families, management strategies, and available support networks. Please check it out and let us know if there are any topics you’d like us to expand on or add.
Drug Repurposing Study: We are involved in an innovative research project that aims to identify FDA-approved drug candidates that could be repurposed to treat ADLD. If a favorable drug emerges, it could help reduce the effects of the disease and improve patient quality of life.