They Created a Drug for Susannah: What About Millions of Other Patients?
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Susannah Receives Personalized Medicine
Among the three patients treated this fall is Susannah, a beautiful 8 year old girl who suffers from a neurodegenerative nano-rare condition. Susannah is receiving a personalized, experimental ASO discovered and developed for her unique mutation.
3 minutes
Most people want to do meaningful work and want to be a part of an organization of which they are proud
Back to Stan’s Space 50 Lessons After [...]
Most people are more limited by their fears than their abilities.
Back to Stan’s Space 50 Lessons After 50: [...]
CROs and Their Role for Nano-rare
with Jamie Macdonald
Cardiovascular Part 2B: (The Pump) Heart Diseases
Diseases of [...]
Cardiovascular Part 2A: (The Pump) Heart Anatomy
Why does [...]
Evaluating human mutation databases for ‘Treatability’ using personalized antisense oligonucleotides
Mittal, S., Tang, I., Gleeson, J.G.
Shifting Mindsets to Expedite Rare Treatments
with Dr. Emil Kakkis
