Shifting Mindsets to Expedite Rare Treatments
with Dr. Emil Kakkis
Coming Together for the Nano-rare Patient
with Dr. John Maraganore
Anna’s Story of Hope and Help
with Dr. Neil Shneider and Sonja Kämpfer
Advocating for Rare Patients
with Wendy Erler
Clinical Trials for One Patient
with Dr. Joe Gleeson
A Diagnostic Odyssey: The UDN and n-Lorem’s Roles
with Dr. Francis Sessions Cole, III
Susannah’s Story:
with Dr. Wendy Chung and Luke Rosen
