An Answered Prayer
N-Lorem to the Rescue
N-Lorem accepted Ireland as a patient in 2021. They have made incredible progress on the research and development of a personalized medicine for her. Ireland's program is estimated to cost $1.2 million. N-Lorem is a nonprofit and generously provides their medicines for free, for the life of the patient.
What is an ASO?
Antisense Oligonucleotides (ASOs) are designed to bind precisely with RNA, modifying the process of creating a disease-causing protein. ASOs are short strands of modified DNA that can specifically target the transcripts of a defective gene to modify the product of that gene.
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Why We Are So Hopeful
An ASO is a genetic based treatment that we hope will improve several of her symptoms and has the possibility to slow the progression of her neurodegenerative disease. 7/7 N-Lorem patients show evidence of clinically important benefit with no adverse reactions.
We are very grateful to the CACNA1A Foundation for their support of the development of Ireland's iPSC cells. Those cells were used by N-Lorem to create Ireland's brain cells to test ASOs on.
