Beautiful words

Tonight I found some of the most beautiful words I have ever read and I want to share them with you.

Are you ready? Well, here goes:

“RGX-121 is REGENXBIO Inc.’s product candidate for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. Individuals with MPS II have a deficiency in the lysosomal enzyme iduronate 2-sulfatase (IDS), an enzyme found throughout the body, including in cells in the central nervous system (CNS) that is responsible for the breakdown of polysaccharides. RGX-121 uses the AAV9 vector to deliver the human IDS gene to the CNS creating the potential that normal IDS enzyme can be produced. Delivery of the gene encoding the enzyme that is deficient within cells in the CNS could provide a permanent source of secreted IDS beyond the blood-brain barrier, allowing for long term cross-correction of cells throughout the CNS. This strategy could also provide rapid IDS delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occurs in Hunter syndrome patients. The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to RGX-121 for the treatment of MPS II. For more information, visit

Did you understand all that? Thought not!

It may not seem a very beautiful piece of writing to you, but to me it means the possibility of a cure, the greater possibility of life beyond the teenage years, the possibility of no more weekly infusions.

Anyone need a translation? Basically, it’s one of the ways that is currently being developed to deliver gene therapy. It would get Pudding’s body to start producing its own enzyme. But more excitingly than that, it would halt the progression of disease in his brain too.

It’s still a way off. They won’t be filing a request for a clinical trial until 2017. And it will be a long drawn-out process with its own risks and inclusion criteria. But there’s time and there’s hope. Hope for Pudding and the other boys like him. I’ve known for a while that gene therapy is in the pipeline, but there is something about seeing something like this in print that means so much more.

So look back over those dry clinical phrases, and perhaps you can see how beautiful they seem to me….


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