On February 15, the GM2 Community shared their experiences of living with Tay-Sachs and Sandhoff diseases during the first-ever, Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting with the FDA.
You can watch a recording of the GM2 EL-PFDD Meeting here.
Stay tuned for the summary of this first-ever meeting in the “Voice of the Patient” report in the late spring/early summer.
On Thursday, November 10, 2022, the NTSAD Community will gather at the Royal Sonesta in Boston for the first in-person Imagine & Believe event in three years to commemorate NTSAD’s 65th Anniversary.
We Care for Rare
Jessie Jackson Juvenile GM1 Disease
“Jessie (age 30) loves going on adventures! Like going to Nitro Harley Racing. She is not about to let GM1 keep her from LIVING life!”