Learn more about Hampus, Julia, and Isabella from Sweden who all suffer from late infantile GM1 gangliosidosis, a fatal degenerative lysosomal storage disease.

About the GM1 Gangliosidosis EL-PFDD
The GM1 Externally-led Patient-Focused Drug Development Meeting (EL-PFDD) with FDA was held on Friday, October 14th, 2022 as part of the Cure GM1 annual virtual conference. This meeting’s aim was to shed light on the experiences and perspectives of GM1 gangliosidosis patients and those who care for them. The information gathered will influence the development and evaluation of drugs to treat GM1.

Please DONATE to help support further GM1 research:
http://www.curegm1.org/donate