Ion Channel Mutations: The Complexities of Treatment
Arguably, the [...]
Why We Dose Intrathecally
Intrathecal administration [...]
Kuri’s Story: DRPLA
6 minutes
Behind the ASO Design and Discovery
with Konstantina Skourti-Stathaki, Ph.D.
Best of the 2023 Nano-rare Patient Colloquium
Don't [...]
North American Rare Diseases Summit 2024
July 11, 2024 - Chicago, IL The North America Rare Disease Summit brings together leading researchers, healthcare professionals, patient advocates, policymakers, industry experts, and rare disease patients and families.
The Hurdles of Insuring Patients with Nano-rare Mutations
with Alan Lotvin, M.D.
