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…
Cure GM1 Caregiver Preferences Study has been published
Our Caregiver Preferences study, The caregivers’ assessments of symptom impact and most important symptoms to…
Cure GM1 Foundation and TEGA Therapeutics Establish Collaboration to Develop GM1 Gangliosidosis Therapy
TEGA Cure GM1 Collaboration PDF TEGA WORLD Pres 2023
Two New AAV Gene Therapy Clinical Trials for Infantile and Late Infantile GM1 to Begin in Q1/Q2 2021
Cure GM1 is pleased to share the tremendous news that both Passage Bio and Lysogene have received regulatory approvals…
A Newborn Screening Assay for GM1 Gangliosidosis, Gelb Laboratory, University of Washington
Tandem Mass Spectrometry Assay of Beta-Galactosidase for Multiplex Newborn Screening of GM1 Gangliosidosis Michael H….
First Gene Therapy Clinical Trial Initiated May 2019!
The first gene therapy clinical trial for GM1 Gangliosidosis is a beacon of hope for…
Passage Bio and UPenn Orphan Disease Center AAV Gene Therapy and Natural History Study
Passage Bio was recently launched with the vision to be a first in class fully-integrated…
2019 The Jackson Laboratory – Natural History Studies of Two GM1 Mouse Models
Scientific researchers Cat Lutz and Aamir Zuberi at the Rare Disease Center of the Jackson…
Axovant Intravenous AAV9 Gene Therapy 2019 Clinical Trial Announced!
GM1 Intravenous AAV9 Gene Therapy Clinical Trial to Begin in the First Half of 2019…