Armand’s Story
From Armand’s Mother My son Armand Hayes was diagnosed in 2011. We are doing all…
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Cody’s Story
From Cody’s Mother Our son Cody Butcher is now 3. He was born on the…
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Anisa’s Story
From Anisa’s Mother My last child has GM1. I also have two girls and a…
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Lydia’s Story
From Lydia’s Mother Lydia was born, apparently healthy, a normal pregnancy. She was born with…
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Lois’ Story
From Lois’ Mom: Lois was born in 2006. She was the first girl in the…
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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…
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2018 Jackson Laboratory – Publicly Available Mouse Models
Dr. Cat Lutz and Dr. Aamir Zuberi are scientists at The Jackson Laboratory. Dr. Lutz…
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2017 Dorphan Small Molecule Research and Mouse Study
Cure GM1 granted funds for a drug repurposing project and mouse study to study a…
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2017 UC Davis Lentiviral Gene Therapy Research
Cure GM1 recently granted funds towards the development of a lentiviral gene therapy approach using…