A Newborn Screening Assay for GM1 Gangliosidosis, Gelb Laboratory, University of Washington
Update (October 2026): This assay work led to later publications, including the Gelb Laboratory’s 2023…
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First Gene Therapy Clinical Trial Initiated May 2019!
Update (October 2026): Axovant, later renamed Sio Gene Therapies, no longer exists. The intravenous AAV9…
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Passage Bio and UPenn Orphan Disease Center AAV Gene Therapy and Natural History Study
Update (October 2026): Passage Bio no longer exists. Its GM1 gene therapy, PBGM01, is now…
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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!
Update (October 2026): Axovant, later renamed Sio Gene Therapies, no longer exists. The intravenous AAV9…
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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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2015-2017 Auburn/UMass Research Intravenous AAV9 Gene Therapy
Update (October 2026): This program entered human clinical trials in 2019 through Axovant. Axovant, later…
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Syner-G Clinical Study
Update (October 2026): This study (NCT02030015) was terminated before completion. For current information on miglustat,…
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IntraBio Inc
Update (October 2026): IntraBio’s drug levacetylleucine (AQNEURSA) is now FDA approved for Niemann-Pick disease type…
