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….
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First Gene Therapy Clinical Trial Initiated May 2019!
The first gene therapy clinical trial for GM1 Gangliosidosis is a beacon of hope for…
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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…
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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…
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Axovant Intravenous AAV9 Gene Therapy 2019 Clinical Trial Announced!
GM1 Intravenous AAV9 Gene Therapy Clinical Trial to Begin in the First Half of 2019…
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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…