Denisa’s Story
DENISA’S STORY My Story “Hi, my name is Dana. I have a daughter with Juvenile…
Over $461K Grant toward Enzyme Replacement Therapy with TEGA Therapeutics
TEGA Therapeutics, Inc. has been awarded a Phase I Small Business and Innovative Research (SBIR)…
Gelb Laboratory Publishes Third GM1 Newborn Screening Paper
Cure GM1 provided seed funding for, and families from the GM1 community provided dried blood…
Asia’s Story
This is the story of Asia, a little princess born on December 6, 2020. Asia…
2022 Cure GM1 Impact Report – Impact in 2022 and Beyond
Our summary of impact in 2022 is now available to read. After some time reflecting,…
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…
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…
Sweet Iris’ Story
About Sweet Iris Iris was diagnosed with Juvenile GM1 when she was 5 and a…