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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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
Passage Bio was recently launched with the vision to be a first in class fully-integrated…
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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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Hampus, Julia, and Isabella’s Story
From Hampus, Julia and Isabella’s Dad This is Hampus, Julia and Isabella. Hampus was born…
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Anna Laura’s Story
From Carla, Anna Laura’s Mother: Hi, I’m Carla, Anna Laura’s mother. Anna Laura is also…
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Hannah’s Story
From Hannah’s Mother: We miss our little angel! We are writing our little girl’s story…
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Tayah’s Story
From Tayah’s mother: This is my little Tayah and we want to give her every…
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Sofia’s Story
From Sofia’s Mother: My daughter Sofia, 19 months. Unfortunately, she lost her battle against GM1…