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How you can help provide experimental ASO treatments to patients with ultra-ultra-rare diseases.

Over the last three years, I have worked to create a charitable foundation, n-Lorem, whose mission is to provide experimental ASO treatments to patients with ultra-ultra-rare diseases (n-1-20 or so in the world). I am pleased to say that since we took n-Lorem public, n-Lorem is off to a strong start. We have already helped clinical investigators treat a number of patients and at n-Lorem, we are averaging more than an application for treatment per week and progressing toward treating a significant fraction of the patients who have been submitted.

That it is possible to create a new ASO to treat an individual patient for free for live is quite remarkable. It reflects more than 30 years of scientific effort that created antisense technology and advances in genomics to make such an opportunity possible. All of us involved with n-Lorem are thrilled to be in position to offer hope and treatments to these desperate patients and families.

n-Lorem is, as I said, off to a strong start, but there are many more desperate patients and families in need so there is vastly more to do. The more help we have, the more we can do. If you would like to learn more about n-Lorem, please feel free to contact us.