N-lorem Foundation

This trial tests a personalized drug called nL-TARDB-006, designed for people with ALS caused by a specific genetic mutation in the TARDBP gene. The drug is an antisense oligonucleotide, which aims to target the genetic cause of the disease. Six participants will receive the trea…

This study tests a personalized antisense oligonucleotide drug designed for a single person with ALS caused by a specific TARDBP gene mutation. The drug aims to reduce harmful TDP-43 protein and slow disease progression. The participant's clinical function, survival, and biomarke…

This study tests a personalized drug called an antisense oligonucleotide, designed specifically for people with ALS caused by a particular change in the CHCHD10 gene. The drug aims to slow or stop the disease by targeting the genetic root cause. The trial involves a small group o…

This trial tests a personalized medicine approach for a single person with amyotrophic lateral sclerosis (ALS) caused by a specific mutation in the CHCHD10 gene. The experimental drug, called an antisense oligonucleotide, is designed to target the genetic root of the disease. Res…

This study tests a custom-designed antisense oligonucleotide (ASO) drug for one person with ALS caused by a specific CHCHD10 gene variant. The drug aims to slow disease progression by targeting the genetic root of the condition. Researchers will monitor changes in muscle function…

This study tests a personalized medicine called an antisense oligonucleotide, designed specifically for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic brain disorder. The treatment aims to reduce seizures and improve quality of life by targeting the …

This study tests a personalized medicine called an antisense oligonucleotide, designed specifically for one person with autosomal dominant leukodystrophy (ADLD), a rare genetic brain disease. The treatment aims to slow or stop the disease by targeting the underlying genetic mutat…

This study tests a custom-made drug for one child with a rare, severe brain disease called CONDBA, caused by a specific gene mutation. The drug aims to slow or stop brain damage by targeting the faulty gene. Researchers will track changes in movement, coordination, and quality of…

This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic disorder that affects movement and brain function. The drug aims to reduce the harmful effects of the mutated AT…

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. Only one participan…

This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with Charcot-Marie-Tooth disease type 2D (CMT2D) caused by a specific GARS1 gene mutation. The drug aims to improve motor skills and quality of life. Only one participant is enro…

This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed specifically for one person with Bainbridge-Ropers syndrome, a rare genetic condition that causes developmental delays. The drug aims to correct the effects of a specific ASXL3 gene mutation. T…

This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed specifically for one person with a rare genetic brain disorder (NEDBA) caused by a MAPK8IP3 mutation. The goal is to see if the drug can improve motor skills and reduce seizures over 12 to 24 m…

This study tests a custom-made drug called an antisense oligonucleotide, designed specifically for one person with retinal dystrophy caused by a PRPH2 gene mutation. The drug aims to correct the genetic error and potentially slow vision loss. The trial involves only one participa…