N-lorem Foundation

This study tests a custom-made drug (called an antisense oligonucleotide) designed specifically for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic brain disorder. The drug targets the faulty ATN1 gene to potentially slow or improve movement problems,…

This study tests a personalized medicine called an antisense oligonucleotide (ASO) designed for one child with a rare genetic brain disease (CONDBA) that causes brain shrinkage and movement problems. The goal is to see if the drug can improve or stabilize motor skills, coordinati…

This study tests a personalized medicine designed for one person with a rare genetic brain disorder (NEDBA) caused by a MAPK8IP3 mutation. The treatment is an antisense oligonucleotide (ASO) that aims to improve motor skills and reduce seizures. The single participant will be fol…

This study tests a custom-made drug for one person with Charcot-Marie-Tooth disease type 2D, a rare genetic nerve condition that causes muscle weakness. The drug is designed to target the specific genetic error causing the disease. The goal is to see if it can improve motor skill…

This study tests a custom-made genetic drug (antisense oligonucleotide) designed for one person with retinal dystrophy caused by a specific PRPH2 gene mutation. The goal is to see if the drug is safe and can slow or stop vision loss. Only one participant will receive the treatmen…

This study tests a custom-made genetic drug (called an antisense oligonucleotide) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare inherited brain disease. The drug aims to reduce seizures and improve quality of life. Only one participant is enrol…

This study tests a custom-made genetic drug (antisense oligonucleotide) designed for one person with Bainbridge-Ropers syndrome caused by a specific ASXL3 gene change. The goal is to see if the drug is safe and can improve motor skills over two years. Because the treatment is per…