Cure GM1 to Collaborate with xCURES for Natural History Study Pilot
Update (October 2026): This pilot has ended. Medical records from 4 patients were collected through…
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Over $461K Grant toward Enzyme Replacement Therapy with TEGA Therapeutics
Update (October 2026): Cure GM1’s collaboration with TEGA Therapeutics has ended, and the Cure GM1…
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Gelb Laboratory Publishes Third GM1 Newborn Screening Paper
Cure GM1 provided seed funding for, and families from the GM1 community provided dried blood…
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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,…
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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…
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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…
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Cure GM1 Caregiver Preferences Study has been published
Our Caregiver Preferences study, The caregivers’ assessments of symptom impact and most important symptoms to…
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Cure GM1 Foundation and TEGA Therapeutics Establish Collaboration to Develop GM1 Gangliosidosis Therapy
Update (October 2026): Cure GM1’s collaboration with TEGA Therapeutics has ended, and the Cure GM1…
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Two New AAV Gene Therapy Clinical Trials for Infantile and Late Infantile GM1 to Begin in Q1/Q2 2021
Update (October 2026): Lysogene, Sio Gene Therapies (formerly Axovant) and Passage Bio no longer exist….
