Why We Dose Intrathecally

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/