Florence’s Story
Florence has late infantile GM1. She was diagnosed when she was 2. She has real…
Sara’s Story
A Message from Sara’s Mother Hello Friends and Families We are an affected family from…
Maria Eduarda’s Story
Maria Eduarda was born on 07/07/2015. She was diagnosed at one year of age with…
T-Jay’s Story
In these images, T-Jay is nearly 18 months old. He was diagnosed with GM1…
Axovant Intravenous AAV9 Gene Therapy 2019 Clinical Trial Announced!
GM1 Intravenous AAV9 Gene Therapy Clinical Trial to Begin in the First Half of 2019…
Lois’ Story
From Lois’ Mom: Lois was born in 2006. She was the first girl in the…
2018-2020 UCSF Preclinical development of a gene therapy for GM1 Gangliosidosis
Widespread vector distribution in the brain and vector serotype selection have presented significant challenges to…
2018 Jackson Laboratory – Publicly Available Mouse Models
Dr. Cat Lutz and Dr. Aamir Zuberi are scientists at The Jackson Laboratory. Dr. Lutz…
2017 Dorphan Small Molecule Research and Mouse Study
Cure GM1 granted funds for a drug repurposing project and mouse study to study a…
2017 UC Davis Lentiviral Gene Therapy Research
Cure GM1 recently granted funds towards the development of a lentiviral gene therapy approach using…