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…
-
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…
-
Hannah’s Story
From Hannah’s Mother: We miss our little angel! We are writing our little girl’s story…
-
Tayah’s Story
From Tayah’s mother: This is my little Tayah and we want to give her every…
-
Sofia’s Story
From Sofia’s Mother: My daughter Sofia, 19 months. Unfortunately, she lost her battle against GM1…
-
Toby’s Story
From Toby’ Mother: On March 21st, 2015, a beautiful baby boy was delivered weighing a…
-
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…