March 25-27, 2023, over 60 doctors, researchers, ALSP patients and caregivers gathered at the University of California, Irvine to partner and address challenges facing ALSP patients.
Four goals were given at the beginning of the conference to set the tone and maintain focus throughout the weekend.
*Better understand the challenges patients face along their diagnostic journey.
*Support clinicians in their goal to treat ALSP and other leukodystrophies.
*Connect patient advocacy groups with funding agencies to build roadmaps to novel treatment options for patients.
*Foster and build relationships between science researchers and physicians so that research can match clinical needs.
To begin the process of achieving these goals, a few key things happened at the conference. First, many insightful scientific presentations were given. Topics covered included an overview of CSF1R related leukoencephalopathy, advancement in the care of ALSP patients, HSCT risks and benefits, ALSP diagnostic challenges, and novel treatments for ALSP such as cell-based therapies and microglia replacement.
These presentations acted as the foundation for all of the following conversations. Time was specifically set aside during meals, breaks, and mingling for doctors, researchers, patients, and caregivers to come together for continued communication. It was in these conversations that pieces began to fit together in building the tools needed to meet the stated goals. By meeting and seeing ALSP patients and caregivers, clinicians and researchers heard first hand the difficulties of living with ALSP.
We are pleased with the connections formed and the progress made during the conference. Many thanks to the California Institute for Regenerative Medicine (CIRM) and the University of California, Irvine for making this event possible.