n-Lorem delivers HOPE and treatment to patients with ultra-rare diseases.

Описание к видео n-Lorem delivers HOPE and treatment to patients with ultra-rare diseases.

n-Lorem Foundation is the first and only non-profit organization to charitably provide personalized antisense oligonucleotide (ASO) treatments to patients with ultra-rare diseases (1-30 patients, worldwide) – for free, for life.

Connor Dalby was born with a mutation that is unique to him. No other person in the world has his mutation, which causes severe seizures, movement disorders and severe developmental delays. Only n-Lorem is poised to help by creating an ASO treatment that is unique to Connor and providing ASO to Connor and his family for free, for life.

Ultra-rare diseases destroy not just a patient, but families and Connor’s story is sadly common. Kelley, Connor’s mother eloquently describes how n-Lorem has turned hopelessness to hope by creating a medicine specifically for Connor.

Connor is not alone, but is representative of n-Lorem achieving its mission to bring individualized therapies for the many patients like Connor. We are proud of the progress we have made and can do even more, for more patients with your help.
We are using a charitable model to solve what a commercial model cannot. We can’t do it alone. The more help we have and the more money we raise, the more lives we can save – together. To learn more about the n-Lorem Foundation, visit www.nlorem.org, and follow us on Twitter, Facebook and LinkedIn.

About n-Lorem Foundation
The n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat patients with ultra-rare diseases (1 to 30 patients) that are the result of a single genetic defect unique to only one or very few individuals. ASOs are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO, and executive chairman of the board of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics.

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