Drug Delivery Mechanisms for SMA

Dr. Charlotte Sumner explains how drugs to treat SMA need to be delivered directly to the central nervous system, bypassing the blood-brain barrier.

The big challenge often is that the nervous system has a barrier that prevents many substances from passing into it, the so-called blood brain barrier. And the blood brain barrier is enormously helpful to us actually because it prevents infectious organisms and things from accessing the nervous system and that's a good thing in general. But it makes it a real challenge at times when developing drugs or novel therapeutics because many of these don�€™t naturally pass across the blood brain barrier. Of course there are ways we can get around that. We can actually deliver therapeutics directly to the nervous system, but it is more challenging. One has to put pumps into the nervous system or other sorts of devices to bypass the blood brain barrier and that of course is a bigger challenge.

Spinal muscular atrophy, SMA, RNA, mRNA, splicing, gene, genetic, DNA, antisense, motor neuron, splice, Therapy, Intrathecal, delivery, drug, mechanism, central nervous system, spinal cord, epidural, inject, needle, cerebrospinal fluid, blood-brain barrier, vertebra, axon, dendrite, Hopkins, john Hopkins,

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