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ID 16943

Timing of SMA Therapies

Dr. Charlotte Sumner describes how early intervention is crucial when treating SMA.
I think another really important issue when considering a degenerative disorder like we're talking about in spinal muscular atrophy is the importance of trying to deliver in a timely fashion. That may relate to how of the neurons had been lost at the time that the treatment is delivered. We likely need to get in there at a time when neurons maybe dysfunctional but not all together gone. It may be quite difficult to reverse disorder once motor neurons have been lost in a profound way. So a very important issue for considering how best to do clinical trials with some of these very exciting new treatments is to consider trying to do things at least initially early in this disease. It prevents those motor neurons that are still there from dying and that would be an enormous benefit. So then you could expect that you would prevent progression of the disease, worsening of the disease over time because you arrest the ongoing loss of motor neurons. But it is also very possible that delivery even when motor neurons have already been lost will be very helpful for those motor neurons that remain. And maybe even allow them to function even better and make muscle a little stronger.
Spinal muscular atrophy, SMA, RNA, mRNA, splicing, gene, genetic, DNA, antisense, motor neuron, splice, Therapy, intervention, degenerative, Hopkins, john Hopkins,
Creative Commons License This work by Cold Spring Harbor Laboratory is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 3.0 United States License.

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Dr. Sumner talks about gene therapy for SMA.
16931. 2D Animation of Genes and Inheritance
An animation introduces DNA and the pattern of inheritance for SMA.
16954. Clinical Trials for New SMA Therapies
Dr. Sumner discusses the clinical trial process for SMA antisense oligonucleotide therapy.
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