Eve’s Story
From Eve’s Mother We live in Ukraine in the Odessa region. Eve was born…
A Newborn Screening Assay for GM1 Gangliosidosis, Gelb Laboratory, University of Washington
Tandem Mass Spectrometry Assay of Beta-Galactosidase for Multiplex Newborn Screening of GM1 Gangliosidosis Michael H….
Sing Me a Story
We are pleased to announce a special nonprofit partnership with the Sing Me a Story…
Pia’s Story
From Pia’s Mother: Hello, my name is Stefany Sanchez. I am 23 years old. My…
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…