Shifting Mindsets to Expedite Rare Treatments
with Dr. Emil Kakkis
Cardiovascular System Part 1: Blood
That warm [...]
Coming Together for the Nano-rare Patient
with Dr. John Maraganore
Antisense (How we do it at n-Lorem)
How we [...]
Anna’s Story of Hope and Help
with Dr. Neil Shneider and Sonja Kämpfer
Why ASO Technology?
What makes [...]
Advocating for Rare Patients
with Wendy Erler
Drug Discovery Platforms
There are [...]
Clinical Trials for One Patient
with Dr. Joe Gleeson
How Drugs are Used
Drugs are [...]
