What do you think about Gene Therapy?
On July 12th I started QALSody/Tofersen treatments. I just finished treatment number 2 today.
Tofersen/QALSody is the first treatment that is designed to target a mutated gene that is responsible for approximately 1-2% of ALS cases.
The scientific explanation for how QALSody stops or prevents ALS in people with a SOD1 mutation is, “Qalsody is an RNA-based therapy designed to prevent the toxic SOD1 protein from being made. It works by binding to and promoting the degradation of SOD1 mRNA molecules, which are the intermediate molecules produced from the gene that are used as a template for protein production.”
“Tofersen (BIIB067) is an antisense oligonucleotide (ASO) that facilitates the degradation of SOD1 messenger RNA to diminish SOD1 protein production.” In some cases, they have seen people in the clinical trials actually gain some things back!
This is a huge step in the right direction for finding treatments for ALS. They are researching treatments for other mutations. They have also identified mutations, including the SOD1 gene, in many cases of sporadic ALS.
What are your thoughts on this treatment and research?
If you are interested in knowing more about my ongoing treatments and how it is affecting my ALS, just let me know and I’d be happy to share.
Corrected and noted source
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