N-lorem Foundation

This study tests a personalized medicine designed for a single person with retinal dystrophy caused by a specific PRPH2 gene mutation. The treatment is an antisense oligonucleotide (ASO) given to try to slow or stop vision loss. The main goals are to check safety and see if the d…

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

This study tests a custom-made drug (called an antisense oligonucleotide) designed for one person with Bainbridge-Ropers syndrome, a rare genetic disorder. The drug aims to correct the effects of a specific gene error. The main goals are to check if the treatment is safe and if i…

This study tests a personalized medicine for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic brain disease that affects movement and thinking. The drug is an antisense oligonucleotide designed to target the faulty ATN1 gene. Researchers will track cha…

This study tests a custom-made drug designed for one person with Charcot-Marie-Tooth disease type 2D, a rare genetic nerve condition that causes muscle weakness. The drug, called an antisense oligonucleotide, aims to correct the effects of a specific GARS1 gene mutation. The sing…

This study tests a custom-made genetic medicine (called an antisense oligonucleotide) designed specifically for one child with a rare, inherited brain disease that causes brain shrinkage and loss of movement. The goal is to see if the drug can slow or improve motor skills and qua…

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…