Fatima and Sultan
From Sultan and Fatima’s mother: I am Sultan Ali and Fatima Shoaib Ali’s mother. …
Eva’s Story
Eva Maria 15.01.16-31.05.19
First Gene Therapy Clinical Trial Initiated May 2019!
The first gene therapy clinical trial for GM1 Gangliosidosis is a beacon of hope for…
Vitto’s Story
Vitto’s Story from his family He was born on May 22, 2018 at 4AM in…
Leonor’s Story
Hello I am Sao Piteira and we are from Portugal. I am the Leonor’s mother…
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…
Brayden’s Story
From Brayden’s Mom: Brayden was born on November 16, 2017 First Symptoms On Tuesday Feb…
Joey and Oliver’s Story
Joey, diagnosed with Juvenile GM1 on April 21, 2017 Oliver, asymptomatic, but diagnosed with Juvenile…
Hampus, Julia, and Isabella’s Story
From Hampus, Julia and Isabella’s Dad This is Hampus, Julia and Isabella. Hampus was born…
Anna Laura’s Story
From Carla, Anna Laura’s Mother: Hi, I’m Carla, Anna Laura’s mother. Anna Laura is also…