J
Jo Ng
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Sep 13, 2026
Ep. 218: Beyond Symptom Control: Gene Therapy for Rare Neurological Disorders
14:56
15:06
15:16
K
14:49Kleopas KleopaMODERATOR
drawbacks that we have experienced?

Jo NgGUEST
if we think more, I think now the world has much more experience of Zolgensma gene therapy, so the gene therapy for SMA.

Jo NgGUEST
So that, you know, was approved in 2017 and I believe over 3,000 patients have been dosed with Zolgensma.

Jo NgGUEST
So there is key things about SMA that time is neuron and timely diagnosis and the therapeutic window for Zolgensma is really key on how effective and the prognosis of these patients are so I mean I think in the US an SMA diagnosis is treated like a genetic emergency so they are fortunate to have newborn screening and they there is significant pressure to dose you know, babies within, you know, a few months of age.
K
19:52Kleopas KleopaMODERATOR
So do you think that any new technologies will probably help to improve safety, improve the targeting, reduce, you know, eventually reduce the cost? How do you see all these emerging technologies?