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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T-Jay’s Story
In these images, T-Jay is nearly 18 months old. He was diagnosed with GM1…
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Maria Eduarda’s Story
Maria Eduarda was born on 07/07/2015. She was diagnosed at one year of age with…
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Sara’s Story
A Message from Sara’s Mother Hello Friends and Families We are an affected family from…
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Florence’s Story
Florence has late infantile GM1. She was diagnosed when she was 2. She has real…
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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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Lois’ Story
From Lois’ Mom: Lois was born in 2006. She was the first girl in the…
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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…