Seeing with Grace: A Teen’s Journey with a Rare Vision-Affecting Disease
[...]
Sneak Peek of the 2025 Nano-rare Patient Colloquium
Brady Huggett interviews Stan Crooke
Meet Rescue 7: Firefighters Serving Patients
with Luke Rosen, Raena and Frank Vrtochnick
Hope for Kinsley: Facing TUBB4A with Courage and Care
with Sarah and Ryan Marta
New Insights into Genetically Caused Developmental Delays
Emerging [...]
Commitment to Safety First: The DSMB
with Eugene Schneider, M.D.
Connor’s Story: A First for TUBB4A Treatment
with Diana and Mike Gooley
