Diego’s Story
Diego was born on June 29, 2021 in Imola, a small town in Italy. We…
The Voice of the Patient Report from the GM1 Gangliosidosis Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD)
You can now download and read the touching and impactful summary of input from GM1…
About the GM1 Gangliosidosis Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting with FDA
The GM1 Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD) was held on Friday, October 14 as…
The February 2023 Cure GM1 Catalyst
Rare Disease Day is on February 28, 2023. Leading up to the day, there are…
Rare Disease Day Photo Contest Entry Form
The 2023 Cure GM1 RDD Photo Contest Calling all photographers! Photo submissions for the 2023…
Cure GM1 Caregiver Preferences Study has been published
Our Caregiver Preferences study, The caregivers’ assessments of symptom impact and most important symptoms to…
The January 2023 Cure GM1 Catalyst
Thank You Thank you for your support in 2022! Our mission to help develop effective…
Strahinja’s Story
Hello, I’m Strahinja, 19 months old from Switzerland. I have two older brothers who are fortunately…
Theodora’s Story
Theodora was born in October 2021. She is a very happy and smiley baby. She…
Camden’s Story
Camden is a happy child. He loves people. Hearing certain names lights his face up….