Fostering Collaboration to Fight ALS
with Manish Raisinghani
Everybody Lives
with Dan Doctoroff
CROs and Their Role for Nano-rare
with Jamie Macdonald
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 Schneider and Sonja Kaempfer
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
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