The Building Blocks of What’s Possible
with David Butler, Ph.D.
Adopting a Rare Son
with Paul Compton
Ion Channel Mutations: The Complexities of Treatment
Arguably, the [...]
Why We Dose Intrathecally
Intrathecal administration [...]
Behind the ASO Design and Discovery
with Konstantina Skourti-Stathaki, Ph.D.
Best of the 2023 Nano-rare Patient Colloquium
Don't [...]
The Hurdles of Insuring Patients with Nano-rare Mutations
with Alan Lotvin, M.D.
Anna’s Story of Hope and Help: FUS-ALS
with Sonja Kämpfer and Dr. Neil Shneider
Mostyn’s Story: Mother’s Day Special
with Zoe Hummel
