Patient Empowerment Program: A Rare Disease Podcast

Why We Dose Intrathecally


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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/

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Patient Empowerment Program: A Rare Disease PodcastBy n-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen)

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