Treatments for Spinal Muscular Atrophy
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It has completely changed what we've seen in the past with infant with SMA, where by the age of two, they're completely reliant on breathing treatments, breathing machines to help them breathe and, um, feeding tubes to help them eat, to now an almost if not normal infant if it were to be administered fairly early on. One good thing with gene therapy is in some ways, it's considered to be a curative treatment, right? So it is a one-time administration. And our hope is that it completely change your gene, and you won't have to get any additional treatment.
So the second therapy, uh, that has been approved now for over a couple of years also consider in some ways a gene-modifying therapy. It's given through your spinal column, and it's given about one every four months. It's approved to use for all types of SMA, and what it does that it helps to target certain region of your gene to help to produce, uh, the protein that's needed to correct the-- the condition of SMA. Both of them have extremely promising results and will forever change the natural history of SMA.
HAN PHAN
We are living in a very exciting time in neuromuscular community. Um, there are now two approved treatments for our patients with spinal muscular atrophy. One of them is a gene therapy treatment, um, and the thought of it is that if you are able to intervene early on, you can hopefully halt the condition or slow down the progression of the condition. So gene therapy has been approved for patients with type 1 SMA. It has completely changed what we've seen in the past with infant with SMA, where by the age of two, they're completely reliant on breathing treatments, breathing machines to help them breathe and, um, feeding tubes to help them eat, to now an almost if not normal infant if it were to be administered fairly early on. One good thing with gene therapy is in some ways, it's considered to be a curative treatment, right? So it is a one-time administration. And our hope is that it completely change your gene, and you won't have to get any additional treatment.
So the second therapy, uh, that has been approved now for over a couple of years also consider in some ways a gene-modifying therapy. It's given through your spinal column, and it's given about one every four months. It's approved to use for all types of SMA, and what it does that it helps to target certain region of your gene to help to produce, uh, the protein that's needed to correct the-- the condition of SMA. Both of them have extremely promising results and will forever change the natural history of SMA.
