n-Lorem Foundation
n-Lorem is a nonprofit that develops individualized ASO treatments for people with extremely rare genetic conditions, at no cost to families. An ASO (antisense oligonucleotide) is a short, custom-made strand of genetic material designed to act on one specific gene change.
n-Lorem is currently considering applications from families with dominant ReNU2. Recessive ReNU2 is not being accepted at this time. ReNU2 Foundation shares this as information only. We do not endorse n-Lorem and have no financial relationship with it.
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Start by reading n-Lorem's Qualifications for Treatment page. If your family meets the criteria, use the "Inquire" button on that page to fill in the Pre-Application Submission Inquiry Form. n-Lorem reviews each inquiry and will tell you whether your family member may be a candidate.
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n-Lorem's mission is to provide individualized ASO medicines to people with "nano-rare" genetic conditions, meaning conditions shared by very few people worldwide. Each treatment is made for a specific gene change and given under the care of a research physician.
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n-Lorem can currently treat only patients who live in the United States.
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According to n-Lorem, a patient must:
Live in the United States
Have a confirmed genetic diagnosis, ideally with full genome analysis
Have a gene change whose effect is understood, which usually means being seen by a geneticist
Be cared for by an experienced research physician at a hospital or academic medical center able to support an individual research treatment plan with the FDA (called a Research IND)
n-Lorem has said that families with dominant ReNU2 may apply, even though more people have dominant ReNU2 than n-Lorem's usual "nano-rare" limit.
n-Lorem notes that ASO technology has limits and it may not be able to help every patient.
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n-Lorem provides its treatments free of charge, for life.
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n-Lorem's own answers: Pre-Application Submission FAQs (PDF)
