Why We Dose Intrathecally
Patient Empowerment Program: A Rare Disease Podcast
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Show Notes
The affected organs of our patients determine the route of administration for an ASO, as certain routes can better target specific organs and offer the best potency. The central nervous system (CNS) is the most common target we encounter, and for this, we dose intrathecally. Let’s dive into how this is done and the particulars of why this approach is used.
The Human Body and Barriers
The Four Tubes – Enteral, Respiratory, Urinary, and the Central Nervous System (CNS) tube
How The Body Constructs Barriers
What is Cerebrospinal Fluid (CSF)
The Blood-Brain Barrier (BBB)
The Types of Molecules That Are Admitted to and Excluded From the CNS
Intrathecal Administration of Drugs
Outwitting the Blood-Brain Barrier
Survey Link – Patient Empowerment Program Podcast: https://forms.office.com/r/1ik9WNs7QB
Register for the 2024 Nano-rare Patient Colloquium: https://www.nlorem.org/nano-rare-patient-colloquium-2024/