Fatima and Sultan
From Sultan and Fatima’s mother: I am Sultan Ali and Fatima Shoaib Ali’s mother. …
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Eva’s Story
Eva Maria 15.01.16-31.05.19
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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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Vitto’s Story
Vitto’s Story from his family He was born on May 22, 2018 at 4AM in…
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Leonor’s Story
Hello I am Sao Piteira and we are from Portugal. I am the Leonor’s mother…
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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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Brayden’s Story
From Brayden’s Mom: Brayden was born on November 16, 2017 First Symptoms On Tuesday Feb…
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Joey and Oliver’s Story
Joey, diagnosed with Juvenile GM1 on April 21, 2017 Oliver, asymptomatic, but diagnosed with Juvenile…
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Hampus, Julia, and Isabella’s Story
From Hampus, Julia and Isabella’s Dad This is Hampus, Julia and Isabella. Hampus was born…